[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Ipsen\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":482},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,22,0,[8,38,62,82,107,129,146,162,181,202,223,244,269,287,309,329,348,370,394,414,436,458],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":21,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":28,"startDateStruct":31,"completionDateStruct":33,"leadSponsor":35,"locationsCount":4},"100640970","a-study-to-observe-the-long-term-safety-of-odevixibat-in-patients-with-alagille-syndrome-algs-who-are-receiving-ongoing-treatment-100640970",false,"NCT07585097","A Study to Observe the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment","Prospective Non-Interventional Study Evaluating the Long-term Safety of Odevixibat in Patients With Alagille Syndrome (ALGS)","Inclusion Criteria:\n\n* Diagnosed with ALGS.\n* On (or starting) active odevixibat treatment.\n* Aged 6 months or older at the time of consent.\n\nExclusion Criteria:\n\n* Currently participating in a clinical trial with odevixibat.\n* Currently participating in any interventional clinical trial for ALGS.\n* Have any contraindication to odevixibat as per the locally approved label.","ALL","6 Months",{"count":19,"type":20},30,"ESTIMATED","2 Years","OBSERVATIONAL","This study will collect information from patients with ALGS who are using odevixibat in their daily lives. Odevixibat is a medication that helps patients with ALGS, a rare disease that affects the liver and causes itching.\n\nThe main aim of this study is to observe the long-term, everyday safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.",[25],"Alagille Syndrome","NOT_YET_RECRUITING","2026-07-01",{"date":29,"type":30},"2026-07-02","ACTUAL",{"date":32,"type":20},"2026-09-30",{"date":34,"type":20},"2031-09-30",{"name":36,"class":37},"Ipsen","INDUSTRY",{"id":39,"slug":40,"hasResults":11,"nctId":41,"briefTitle":42,"officialTitle":43,"acronym":4,"eligibilityCriteria":44,"healthyVolunteers":11,"sex":45,"minAge":4,"maxAge":4,"enrollmentInfo":46,"targetDuration":48,"studyType":22,"phases":4,"briefSummary":49,"conditions":50,"keywords":52,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":56,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":61,"locationsCount":4},"100635763","elafibranor-pregnancy-surveillance-program-a-study-to-evaluate-the-safety-of-elafibranor-during-pregnancy-100635763","NCT07556913","Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy","Elafibranor Pregnancy Surveillance Program: A Post Marketing, Long Term, Multi-Center, Noninterventional, and Descriptive Study to Characterize Pregnancy and Maternal Complications and to Describe Effects on the Developing Fetus, Neonate and Infant Among Individuals Exposed to Elafibranor During Pregnancy","Inclusion Criteria:\n\n* Maternal exposure to at least one dose of elafibranor, either: Within three weeks prior to conception (based on the estimated date of LMP) or At any time during pregnancy (from the estimated date of conception through pregnancy outcome).\n* Participants who were previously enrolled in this program during a past pregnancy and who meet inclusion criteria #1 for the subsequent pregnancy are eligible to enroll again.\n* Informed consent or IRB\u002FIEC-approved waiver of informed consent (not applicable if reported by Ipsen PV according to usual pharmacovigilance practices).\n\nExclusion Criteria:\n\n* Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.","FEMALE",{"count":47,"type":20},3,"33 Months","The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period \\[LMP\\]).\n\nInformation will be collected from participants, their healthcare providers, published studies, and safety databases. Reports of pregnancy linked to elafibranor from clinical trials, spontaneous reports, or literature will also be included, with steps taken to avoid duplicates.\n\nThe study begins once the first participant is enrolled and ends after the last mother and child data are collected. It is planned to run for about 10 years, with infant follow-up lasting up to 2 years, for a maximum total duration of 12 years and 9 months.\n\nThe program is strictly observational. All medical care, visit schedules, and treatment decisions remain with healthcare providers. Only routine medical record data will be collected, and no extra tests or procedures are required.\n\nParticipation is voluntary, and written informed consent will be obtained before enrollment.",[51],"Pregnancy Related",[53,54,55],"Rare disease","Primary Biliary Cholangitis","Pregnancy",{"date":29,"type":30},{"date":58,"type":20},"2026-08-15",{"date":60,"type":20},"2039-01-31",{"name":36,"class":37},{"id":63,"slug":64,"hasResults":11,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":4,"eligibilityCriteria":68,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":69,"targetDuration":71,"studyType":22,"phases":4,"briefSummary":72,"conditions":73,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":76,"startDateStruct":77,"completionDateStruct":79,"leadSponsor":81,"locationsCount":47},"100640181","a-study-of-the-effectiveness-safety-and-the-long-term-outcomes-of-participants-with-progressive-familial-intrahepatic-cholestasis-pfic-who-take-odevixibat-bylvay-in-china-100640181","NCT07588880","A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China","Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay).","Inclusion Criteria:\n\n* Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enroll the participant in this registry) by their treating physician\n* On (or starting) active odevixibat treatment (participants can remain in the registry during odevixibat treatment interruptions)\n* Signed informed consent and assent, as appropriate\n\nExclusion Criteria:\n\n* Currently participating in a clinical trial with odevixibat\n* Currently participating in any interventional clinical trial for PFIC\n* Have any contraindication to odevixibat as per the approved label in China",{"count":70,"type":20},20,"5 Years","This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China.\n\nPFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus).\n\nOdevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC.\n\nThe main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.",[74],"Progressive Familial Intrahepatic Cholestasis","RECRUITING",{"date":29,"type":30},{"date":78,"type":30},"2026-04-16",{"date":80,"type":20},"2031-04-30",{"name":36,"class":37},{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":87,"acronym":88,"eligibilityCriteria":89,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":91,"enrollmentInfo":92,"targetDuration":4,"studyType":94,"phases":95,"briefSummary":97,"conditions":98,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":100,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":106},"100622740","phase-3-a-study-to-assess-how-well-and-safely-elafibranor-works-in-adult-participants-with-primary-sclerosing-cholangitis-100622740","NCT07387549","A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis","A Phase III, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis","ELASCOPE","Inclusion Criteria:\n\n* Adults participants aged 18 years or older\n* Confirmed diagnosis of primary sclerosing cholangitis based on standard clinical, biochemical, and imaging criteria\n* Compensated liver disease at screening\n* Stable background therapy, where applicable prior to study entry\n* Women of childbearing potential have to apply during the entire duration of the study a highly effective method of birth control\n* Ability to provide written informed consent and comply with study procedures.\n\nExclusion Criteria:\n\n\\- History or presence of other concomitant chronic liver disease\n\n\\- History of hepatic decompensation, including: i) History of liver transplantation, current MELD 3.0 score ≥12 due to hepatic impairment.\n\nii) Evidence of complications of cirrhosis\n\n* Participants with cirrhosis who are also classified as Child-Pugh B or C based on the Child Pugh score.\n* History of biliary intervention within 60 days prior to the screening period, and\u002For presence of percutaneous drain or bile duct stent at SV.\n* History of bacterial cholangitis, and\u002For participant on antibiotics for prophylaxis of recurrent cholangitis within 60 days prior to the SV.\n* History or any current suspicion of cholangiocarcinoma or hepatocellular carcinoma\n* Known malignancy or history of malignancy within the last 5 years, with the exception of local, successfully treated basal cell carcinoma or in-situ carcinoma of the uterine cervix.\n* Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).\n* Administration of the following medications are prohibited as specified below:\n\n  i) 3 months prior to baseline: norucholic acid, fibrates, seladelpar and glitazones.\n\nii) 3 months prior to baseline: cyclosporine, mycophenolate, pentoxifylline, and chronic systemic corticosteroids (except as part of management of IBD at an ongoing stable dose); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid, or nitrofurantoin).\n\n* Participants who are currently participating in, plan to participate in, or have participated in an investigational drug or medical device study containing active substance within 30 days or five half-lives, whichever is longer, prior to the SV. - Participants with previous exposure to elafibranor.\n* Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) \\>450 msec in males or QTcF \\>470 msec in females for participants without bundle branch block.\n* Significant renal disease,\n* For female participants: known pregnancy, or has a positive serum pregnancy test, or lactating.\n* Regular alcohol intake in excess of the recommended limit of 2 standard drinks per day for men or 1 standard drink per day for women\n* History of alcohol abuse, or other substance abuse within 1 year prior to SV.\n* Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that contraindicates participation in the study.\n* Mental instability or incompetence\n* Participant has or is known to have tested positive for human immunodeficiency virus (HIV) type 1 or 2 at SV.\n* Medical conditions that may diminish life expectancy to \\\u003C2 years.","18 Years","75 Years",{"count":93,"type":20},350,"INTERVENTIONAL",[96],"PHASE3","The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and serious health problems, including the need for a liver transplant and death.\n\nIn this study, about 350 participants with large duct PSC will take part. Participants will be randomized to receive either elafibranor 120 mg once daily or a placebo (a tablet with no active medicine). The study includes a screening period, an treatment period, and a post-treatment safety follow-up.\n\nDuring the study, participants will undergo routine clinical assessments, laboratory testing, imaging evaluations, and complete patient-reassessments to evaluate liver disease progression, symptoms, quality of life and safety.\n\nFollowing the end of treatment, participants will complete a safety follow-up period at approximately four weeks. Participants may withdraw from the study at any time. Each participant may be in the study for several years, as the treatment period will continue until the study reaches enough health events among participants, which is expected to take about 5 years.",[99],"Primary Sclerosing Cholangitis",{"date":29,"type":30},{"date":102,"type":30},"2026-05-14",{"date":104,"type":20},"2031-05-31",{"name":36,"class":37},4,{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":113,"eligibilityCriteria":114,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":115,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":117,"conditions":118,"keywords":120,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":122,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":128},"100607680","a-study-to-assess-the-genetic-variations-in-bile-flow-disorders-linking-progressive-familial-intrahepatic-cholestasis-pfic-related-genes-to-symptoms-in-adults-with-recurrent-cholestasis-in-spain-100607680","NCT07191704","A Study to Assess the Genetic Variations in Bile Flow Disorders: Linking Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes to Symptoms in Adults With Recurrent Cholestasis in Spain","Characterization of Progressive Familial Intrahepatic Cholestasis (PFIC)-Related Genes in Adult Patients With Idiopathic Recurrent and Chronic Cholestasis in Spain - REGENIC","REGENIC","Inclusion Criteria:\n\n* Adult patients (≥18 years old) with written informed consent prior to data collection and study procedures.\n* Unexplained recurrent and\u002For chronic cholestasis (idiopathic cholestasis), defined as alkaline phosphatase (ALP) or Gamma-Glutamyl Transferase (GGT) \\> Upper Limit of Normal (ULN).\n* Patients who provide the blood sample for the genetic analysis.\n\nExclusion Criteria:\n\n* Patients with clear and confirmed diagnosed causes of cholestasis, including:\n\n  * Primary Biliary Cholangitis\n  * Primary or Secondary Sclerosing Cholangitis\n  * Obstruction of the bile ducts\n  * Other Liver diseases: cholestasis secondary to hepatocellular injury, viral hepatitis (mainly Hepatitis A virus \\[HAV\\], Hepatitis B virus \\[HBV\\] and Hepatitis C virus \\[HCV\\]), toxic hepatitis (pharmacological; drug-induced liver injury \\[DILI\\]), autoimmune hepatitis; intestinal failure, total parenteral nutrition \\[TPN\\]; Wilson's disease, choledochal cyst, Caroli Syndrome, and thick bile due to haemolysis.",{"count":116,"type":20},150,"Progressive Familial Intrahepatic Cholestasis (PFIC) is a group of inherited conditions that affect how bile moves in the liver, which can lead to serious liver problems. Doctors usually recommend genetic testing for patients with unexplained bile issues-after ruling out more common causes-to better understand the problem. However, there isn't much information on how common these genetic changes are in adults with these liver issues, especially in Spain. This study will observe these genetic changes so that doctors can diagnose the condition more clearly and create personalized treatment plans.\n\nThis study will be conducted in several centers across Spain for 10 months. Each adult participant will take part in a single-day visit where their health information will be collected, and a blood sample will be taken for both routine tests and genetic analysis.",[119],"PFIC - Progressive Familial Intrahepatic Cholestasis",[121],"Cholestasis",{"date":29,"type":30},{"date":124,"type":30},"2025-12-08",{"date":126,"type":20},"2026-07-31",{"name":36,"class":37},10,{"id":130,"slug":131,"hasResults":11,"nctId":132,"briefTitle":133,"officialTitle":134,"acronym":4,"eligibilityCriteria":135,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":136,"targetDuration":137,"studyType":22,"phases":4,"briefSummary":138,"conditions":139,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":140,"startDateStruct":141,"completionDateStruct":143,"leadSponsor":145,"locationsCount":47},"100607235","a-study-of-the-effectiveness-safety-and-the-long-term-outcomes-of-participants-with-progressive-familial-intrahepatic-cholestasis-pfic-who-take-odevixibat-bylvay-in-south-korea-100607235","NCT07185919","A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea","Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay)","Inclusion criteria:\n\n1. Diagnosed with PFIC (all types) who have been prescribed odevixibat (independently of the decision to enrol the participant in this registry) by their treating physician\n2. On (or starting) active odevixibat treatment Note: Participants can remain in the registry during odevixibat treatment interruptions\n\nExclusion criteria:\n\n1. Currently participating in a clinical trial with odevixibat\n2. Currently participating in any interventional clinical trial for PFIC\n3. Have any contraindication to odevixibat as per the approved label in South Korea.",{"count":128,"type":20},"7 Years","This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives. Odevixibat is a medicine that helps people with PFIC, a type of rare disease that makes their liver not work well and causes itching and yellow skin. Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Obevixibat was approved by the Ministry of Food and Drug Safety (MFDS) in South Korea on 23 August 2024.\n\nThis study will collect information to see how well and how safe odevixibat is in the long run for participants in South Korea.",[74],{"date":29,"type":30},{"date":142,"type":30},"2025-10-30",{"date":144,"type":20},"2032-09-30",{"name":36,"class":37},{"id":147,"slug":148,"hasResults":11,"nctId":149,"briefTitle":150,"officialTitle":151,"acronym":4,"eligibilityCriteria":152,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":153,"targetDuration":21,"studyType":22,"phases":4,"briefSummary":154,"conditions":155,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":156,"startDateStruct":157,"completionDateStruct":159,"leadSponsor":161,"locationsCount":128},"100581417","a-study-observing-the-long-term-effectiveness-and-safety-of-odevixibat-bylvay-in-patients-with-alagille-syndrome-algs-who-are-receiving-ongoing-treatment-100581417","NCT06850038","A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing Treatment","Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)","Inclusion Criteria:\n\n* Diagnosed with ALGS.\n* On (or starting) active odevixibat treatment.\n* Signed informed consent and assent, as appropriate. Consent\u002Fassent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves.\n\nExclusion Criteria:\n\n* Currently participating in a clinical trial with odevixibat.\n* Currently participating in any interventional clinical trial for ALGS.\n* Have any contraindication to odevixibat as per the locally approved label.\n* Had liver transplant before enrolment",{"count":19,"type":20},"This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.\n\nOdevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching.\n\nThe main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.",[25],{"date":29,"type":30},{"date":158,"type":30},"2025-04-22",{"date":160,"type":20},"2029-11",{"name":36,"class":37},{"id":163,"slug":164,"hasResults":11,"nctId":165,"briefTitle":166,"officialTitle":167,"acronym":168,"eligibilityCriteria":169,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":170,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":172,"conditions":173,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":174,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":180},"100550445","a-study-observing-everyday-effectiveness-and-safety-of-the-drug-elafibranor-in-participants-with-primary-biliary-cholangitis-who-are-receiving-ongoing-treatment-100550445","NCT06447168","A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment","Prospective Non-interventional, Phase IV Multicentre Study to Assess the Effectiveness, Safety and Tolerability of Elafibranor 80 mg\u002FDay in Participants With Primary Biliary Cholangitis Receiving Treatment in a Real-world Setting.","ELFINITY","Inclusion Criteria:\n\n* Participant has provided written informed consent and agrees to comply with the study protocol.\n* Participant with PBC diagnosis.\n* Participant for whom the treating physician has decided to start or participants who are currently receiving treatment with commercialized elafibranor.\n* If a participant has a caregiver who agrees to complete the caregiver questionnaires, an informed consent should be collected from the caregiver before any data is collected.\n\nExclusion Criteria:\n\n* Participant is currently participating or, plans to participate in an investigational drug study or medical device study containing active substance.\n* Participant with known hypersensitivity to the product or to any of its excipients.\n* Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.",{"count":171,"type":20},424,"This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting.\n\nPBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged.\n\nThe liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms including pruritus (itching) and fatigue. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.\n\nIn this study the main aim is to observe the effectiveness, safety and tolerability of elafibranor in participants with PBC who are receiving treatment in real world setting. The total study duration for each participants will be 60 months (approximately 5 years).",[54],{"date":29,"type":30},{"date":176,"type":30},"2024-10-14",{"date":178,"type":20},"2032-07-15",{"name":36,"class":37},68,{"id":182,"slug":183,"hasResults":11,"nctId":184,"briefTitle":185,"officialTitle":186,"acronym":4,"eligibilityCriteria":187,"healthyVolunteers":11,"sex":45,"minAge":4,"maxAge":4,"enrollmentInfo":188,"targetDuration":189,"studyType":22,"phases":4,"briefSummary":190,"conditions":191,"keywords":192,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":195,"startDateStruct":196,"completionDateStruct":198,"leadSponsor":200,"locationsCount":201},"100535982","odevixibat-pregnancy-and-lactation-surveillance-program-a-study-to-evaluate-the-safety-of-odevixibat-during-pregnancy-andor-lactation-100535982","NCT06258902","Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and\u002For Lactation","Odevixibat Pregnancy and Lactation Surveillance Program: A Post-marketing, Long-term, Observational, Descriptive Study to Assess the Risk of Pregnancy and Maternal Complications and Adverse Effects on the Developing Fetus, Neonate, and Infant Among Individuals Exposed to Odevixibat During Pregnancy and\u002For Lactation","Inclusion Criteria:\n\n* Exposure to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and\u002For at any time during lactation (up to 12 months of infant age or weaning, whichever comes first).\n* Informed consent or IRB-\u002FEC-approved waiver of informed consent (not applicable if reported by Albireo PV according to usual pharmacovigilance practices)\n\nExclusion Criteria:\n\n* Refusal to provide informed consent, if required",{"count":70,"type":20},"10 Years","The participants of this study will be of any age who are exposed to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and\u002For at any time during lactation (up to 12 months of infant age or weaning, whichever comes first.\n\nThis study will collect data obtained via a variety of sources, including enrolled pregnant or lactating participants, the healthcare providers (HCP) involved in their care or the care of their infants, if applicable, and Albireo pharmacovigilance.\n\nStudy start date is either start of data collection or first patient enrolled whatever occurs earlier.\n\nThe surveillance program is strictly observational; the schedule of office visits and all treatment regimens are determined by HCPs. Only data that are routinely documented in patients' medical records as part of usual care will be collected.\n\nNo additional laboratory tests or HCP assessments will be required as part of this surveillance program.",[51],[53,193,55,194],"PFIC","Lactation",{"date":29,"type":30},{"date":197,"type":30},"2023-09-28",{"date":199,"type":20},"2032-05-31",{"name":36,"class":37},1,{"id":203,"slug":204,"hasResults":11,"nctId":205,"briefTitle":206,"officialTitle":207,"acronym":208,"eligibilityCriteria":209,"healthyVolunteers":11,"sex":16,"minAge":210,"maxAge":4,"enrollmentInfo":211,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":213,"conditions":214,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":216,"startDateStruct":217,"completionDateStruct":219,"leadSponsor":221,"locationsCount":222},"100522976","a-study-to-document-and-to-further-describe-long-term-safety-and-effectiveness-of-palovarotene-in-participants-with-fibrodysplasia-ossificans-progressiva-fop-100522976","NCT06089616","A Study to Document and to Further Describe Long-term Safety and Effectiveness of Palovarotene in Participants With Fibrodysplasia Ossificans Progressiva (FOP)","An International Observational Registry Study to Further Describe Long-term Safety and Effectiveness of Palovarotene in Patients With Fibrodysplasia Ossificans Progressiva (FOP)","FOPal","Inclusion Criteria :\n\n* Adult or child with FOP who have been prescribed palovarotene (prior to and independently of the decision to enroll the patient in this registry study and as per local label) by their treating physician according to the locally approved product information;\n* Signed informed consent as per local regulations must be obtained and maintained. Consent\u002Fassent from the participant should be obtained as appropriate before any registry study data collection are conducted. If applicable, parents or legally authorized representatives must give signed informed consent.\n\nExclusion Criteria :\n\n* Currently participating in a palovarotene clinical trial;\n* Currently participating in any interventional clinical trial for FOP;\n* Have any contraindication to palovarotene as per the locally approved label (except for pregnant women who have previously received and discontinued palovarotene at any time during the pregnancy and who will be included for safety follow-up).","8 Years",{"count":212,"type":20},100,"The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP).\n\nFOP is an ultra-rare, severely disabling disease characterized by new bone formation in areas of the body where bone is not normally present (heterotopic ossification (HO)).\n\nHO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups).\n\nThis registry study will take place in countries where the treatment, known as palovarotene, has been approved for use. Participants will either be treated with palovarotene (i.e already be receiving palovarotene as prescribed by their treating physician according to locally approved product information) or untreated with palovarotene.\n\nThe main aim of this registry study will be to collect and assess real-world safety data on children and adult participants with FOP treated with palovarotene.\n\nThis registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance.\n\nIn addition, this registry study aims to descriptively compare key safety outcomes (i.e. flare-up episodes, growth outcomes, and bone fractures) between participants exposed and unexposed to palovarotene.",[215],"Fibrodysplasia Ossificans Progressiva",{"date":29,"type":30},{"date":218,"type":30},"2024-12-05",{"date":220,"type":20},"2035-12-05",{"name":36,"class":37},5,{"id":224,"slug":225,"hasResults":11,"nctId":226,"briefTitle":227,"officialTitle":228,"acronym":229,"eligibilityCriteria":230,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":231,"targetDuration":4,"studyType":94,"phases":233,"briefSummary":234,"conditions":235,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":237,"startDateStruct":238,"completionDateStruct":240,"leadSponsor":242,"locationsCount":243},"100517384","phase-3-a-long-term-study-of-elafibranor-in-adult-participants-with-primary-biliary-cholangitis-100517384","NCT06016842","A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis","A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)","ELFIDENCE","Inclusion Criteria :\n\n* Male or female participants must be ≥18 years of age at the time of signing the informed consent.\n* Participants with a definite or probable diagnosis of primary biliary cholangitis (PBC)\n* Participants with cirrhosis at SV1. • Participants must be Child Pugh A or Child Pugh B.\n* Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n\nExclusion Criteria :\n\n* History or presence of other concomitant liver disease including but not limited to:\n\n  * i) Primary sclerosing cholangitis (PSC).\n  * ii) Autoimmune hepatitis (AIH) by simplified Diagnostic Criteria of the International Autoimmune Hepatitis Group (IAIHG) ≥6, or if treated for an overlap of PBC with AIH, or if there is clinical suspicion and evidence of overlap AIH features, that cannot be explained alone by insufficient response to UDCA.\n  * iii) Positive hepatitis B surface antigen (HBsAg). Participants with negative HBsAg and positive hepatitis B core antibody (HBcAb) may be eligible if hepatitis B virus deoxyribonucleic acid (HBV DNA) is negative.\n  * iv) Hepatitis C virus (HCV) infection defined by positive anti-HCV antibody and positive HCV ribonucleic acid (RNA) (Note: Participants with positive anti-HCV antibody due to previously treated HCV infection, may be enrolled if a confirmatory HCV RNA is undetectable and sustained viral response has been documented).\n  * v) Alcohol-associated liver disease (ALD).\n  * vi) Nonalcoholic steatohepatitis (NASH).\n  * vii) Other chronic liver diseases, such as alpha-1 antitrypsin deficiency.\n* History or presence of clinically significant hepatic decompensation, including:\n\n  * i) History of liver transplantation, current placement on a liver transplant list, current model for end-stage liver disease including (MELD) 3.0 score \\>12 due to hepatic impairment.\n  * ii) Evidence of complications of cirrhosis, including hepatic decompensation or evidence of significant portal hypertension complications including presence of uncontrolled ascites; history of variceal bleeding or related interventions (e.g. variceal banding, or transjugular intrahepatic portosystemic shunt placement); presence of hepatic encephalopathy Grade 2 or higher per West-Haven criteria; history or presence of spontaneous bacterial peritonitis. Note: participants with low-risk varices (Grade I) without history of bleeding or other treatment may be eligible to enrol.\n  * iii) Hepatorenal syndrome (HRS) (type I or II ). • vi) Hospitalisation for liver-related complication within 12 weeks prior to SV1.\n* Known history of human immunodeficiency virus (HIV) infection or having a positive confirmatory test for HIV type 1 or 2.\n* Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).\n* Evidence of any other unstable or untreated clinically significant immunological, endocrine, hematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant conditions that are not well controlled.\n* Non-hepatic medical conditions that may diminish life expectancy to \\\u003C2 years, including known cancers.\n* History of hepatocellular carcinoma.\n* Alpha-fetoprotein (AFP) \\>20 ng\u002FmL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of hepatocellular carcinoma.\n* Known malignancy or history of malignancy within the last 5 years. Participants with non-melanoma skin cancer, or carcinoma in situ (e.g. breast carcinoma, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.\n* Administration of the following medications is prohibited during the study, and prior to the study as per the timelines specified below: • i) 3 months prior to baseline: fibrates, seladelpar, glitazones, obeticholic acid, azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid or nitrofurantoin).\n* Participants who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or 5 half-lives, whichever is longer, prior to the screening period.\n\n  i) If the previous study was for an experimental therapy being studied for potential benefit in PBC, and the potential therapeutic agent was proven to have no beneficial effect in PBC and there are no safety concerns, the participant may enrol after 30 days or 5 half-lives from the last dose of the therapeutic agent, whichever is longer.ii) For therapeutic agents being studied for potential benefit in PBC for which it is still unclear if there may be a potential benefit, participants may enrol after 6 months from the last dose of the therapeutic agent.\n* Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) \\>450 msec in males or QTcF \\>470 msec in females for participants without bundle branch block. For participants with bundle branch block or other intraventricular conduction delay, a longer QTcF \\>480 msec would be exclusionary.\n* Total bilirubin (TB) \\>5x ULN\n* Alanine aminotransferase (ALT) and\u002For aspartate aminotransferase (AST) \\>5x ULN at SV1\n* Creatinine phosphokinase (CPK) \\>2x ULN.\n* Platelet count \\\u003C50,000\u002FμL\n* International normalised ratio (INR) \\>1.8 in the absence of anticoagulant therapy.\n* Estimated glomerular filtration rate (eGFR) \\\u003C45 mL\u002Fmin\u002F1.73m2 per the Modification of Diet in Renal Disease (MDRD)-6 Study formula at SV1.\n* Significant renal disease, including nephritic syndrome, chronic kidney disease (CKD) (defined as participants with evidence of significantly impaired kidney function or underlying kidney injury).\n* For female participants: known current pregnancy, or has a positive serum pregnancy test, or is breastfeeding.\n* Participants unwilling or unable to be abstinent from alcohol during the study.\n* History of alcohol abuse, or other substance abuse within 1 year prior to SV1.\n* Known hypersensitivity to elafibranor or to any of the excipients of the investigational product(s).\n* Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.\n* Any other condition that, in the opinion of the investigator, would interfere with study participation or completion, or would put the participant at risk, including a potential participant assessed as being at high risk of noncompliance with the study.\n* Alkaline phosphatase (ALP) ≥10x ULN.\n* Albumin \\\u003C2.8 g\u002FdL due to impaired hepatic function.",{"count":232,"type":20},276,[96],"The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver).\n\nPBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage.\n\nThe liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.\n\nThis study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant.\n\nThe main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death).\n\nThis study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.",[236],"Primary Biliary Cholangitis (PBC)",{"date":29,"type":30},{"date":239,"type":30},"2023-08-31",{"date":241,"type":20},"2029-05-31",{"name":36,"class":37},186,{"id":245,"slug":246,"hasResults":11,"nctId":247,"briefTitle":248,"officialTitle":249,"acronym":4,"eligibilityCriteria":250,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":251,"enrollmentInfo":252,"targetDuration":4,"studyType":94,"phases":254,"briefSummary":256,"conditions":257,"keywords":259,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":263,"startDateStruct":264,"completionDateStruct":266,"leadSponsor":268,"locationsCount":253},"100626905","phase-1-a-study-to-assess-a-medicine-called-tovorafenib-in-japanese-children-and-young-adults-with-brain-tumours-100626905","NCT07441707","A Study to Assess a Medicine Called Tovorafenib in Japanese Children and Young Adults With Brain Tumours","A Phase I, Open-label, Single-arm, Multicentre Study to Evaluate the Safety and Pharmacokinetics of Tovorafenib in Japanese Paediatric Participants With BRAF-altered Recurrent or Progressive Low-grade Glioma","Inclusion Criteria\n\n* Participants must be 6 months to 25 years of age, inclusive, with at least two generations of Japanese ancestry at the time of signing the informed assent\u002Fconsent.\n* Participants must have relapsed or progressive low-grade glioma with a documented known activating BRAF alteration, including BRAF V600 mutations and KIAA1549:BRAF fusions, as identified through molecular assays as routinely performed at Clinical Laboratory Improvement Amendments-certified or other similarly certified laboratories.\n* Participants must have histopathologic verification of malignancy at either original diagnosis or relapse.\n* Participants must have received at least one line of prior systemic therapy and have documented evidence of radiographic progression.\n* Participants must have at least one evaluable and\u002For measurable lesion (imaging must be performed within 28 days of initiation of treatment) as defined by Response Assessment in Neuro-Oncology-high grade glioma criteria (T1 weighted lesion that can be reproducibly measured in at least two dimensions of at least 10 mm, visible on ≥2 axial slices that are preferably, at most, 5 mm apart with 0 mm skip).\n* Participants must have fully recovered from the acute toxic effects of all prior anticancer chemotherapy\n* Chronic toxicities from prior anticancer therapy must be stable and at National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 Grade ≤2; ongoing retinopathy must be ≤1.\n* Participants must have adequate hematologic, hepatic and renal function\n* Participants receiving steroids for tumour-associated symptoms must be on a stable dose (e.g. no initial\u002Floading dose, no increase or decrease) for 14 days prior to C1D1.\n* Participants must be able to swallow tablets or liquid or administer through gastric access via a feeding tube (12 Fr or greater).\n\nExclusion Criteria\n\n* Participant's tumour has an additional previously known or expected to be activating molecular alteration(s) (e.g. histone mutation, isocitrate dehydrogenase 1 and 2 mutations, fibroblast growth factor receptor mutations or fusions, MYBL v-myb avian myeloblastosis viral oncogene homolog-like alterations, neurofibromatosis type-1 somatic or germline mutations).\n* Participant has symptoms of clinical progression without radiographically recurrent or radiographically progressive disease.\n* Participant has known or suspected diagnosis of neurofibromatosis type 1 via genetic testing or current diagnostic criteria.\n* Participant has history of any major disease (e.g. confirmed or suspected diagnosis of interstitial lung disease), other than the primary malignancy under study, that in the opinion of the investigator might interfere with safe protocol participation.\n* Participant has a history or current evidence of central serous retinopathy (CSR), retinal vein occlusion (RVO), or ophthalmopathy present at baseline that would be considered a risk factor for CSR or RVO. Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor or strabismus) will NOT be considered significant abnormalities for the purposes of this study.\n* Participant has major surgery within 14 days (2 weeks) prior to Cycle 1 Day 1 (does not include central venous access, cyst fenestration or cyst drainage, or ventriculoperitoneal shunt placement or revision).\n* Participant has clinically significant active cardiovascular disease, history of myocardial infarction, deep vein thrombosis\u002Fpulmonary embolism within 6 months prior to C1D1, ongoing cardiomyopathy or current prolonged QT interval corrected for heart rate by Fridericia's formula interval \\>470 milliseconds based on triplicate electrocardiogram (ECG) average.\n* Participant has nausea and vomiting NCI-CTCAE v5.0 Grade ≥2, malabsorption requiring supplementation or significant bowel or stomach resection that would preclude adequate absorption of tovorafenib.\n* Participant is neurologically unstable despite adequate treatment (e.g. uncontrolled seizures).\n* Concomitant medications that are strong inhibitors or inducers of CYP2C8 within 14 days before initiation of therapy. Concomitant medications that are substrates of breast cancer resistance protein (BCRP) with a narrow therapeutic index within 14 days before initiation of therapy.\n* Participant has any clinically significant skin toxicity at Screening that in the opinion of the investigator would increase risk of severe skin toxicity when using investigational product.","25 Years",{"count":253,"type":20},6,[255],"PHASE1","The purpose of this study is to evaluate safety and the way the body absorbs, distributes and gets rid of the study drug tovorafenib in the body in Japanese children, adolescents and young adults with specific brain tumours. This includes how the drug is absorbed, distributed and eliminated from the body (called pharmacokinetics). The study will also test how well the drug works to shrink brain tumours.\n\nIn this study, all participants will receive tovorafenib orally once weekly.\n\nThere will be four periods in this study:\n\n1. Screening period (up to 4 weeks): Participants will be evaluated to determine if they can take part in the study, requiring at least one visit to the study centre.\n2. Treatment period (up to 24 months): All eligible participants will receive tovorafenib.\n\n   This requires five visits for the first 2 months (Cycle 1 and Cycle 2) followed by one visit every month (at the start of each treatment cycle). Participants will receive the first oral dose of tovorafenib on Day 1 of Cycle 1 at the study clinic. After Day 1, participants will need to take tovorafenib once weekly on Day 8, Day 15 and Day 22. Participants will be required to come to the study clinic in person at least five times for Cycle 1 and Cycle 2.\n\n   In addition, participants will have one remote visit (telephone call) during Cycle 1. After Cycle 2, only one in-person clinic visit is required at the start of each treatment cycle. A participant will stop treatment if their disease gets worse, if treatment has a harmful effect, or if they do not want to take part in the study anymore.\n3. End-of-Treatment Safety Follow-Up (30 days): Participants will have a clinic visit 30 days after stopping treatment to check their health.\n4. Long-Term Follow-Up (up to 2 years): Participants will be monitored every 3 months unless they start a new anti-cancer treatment or leave the study.\n\nDuring the study, participants will undergo various health measurements and observations, including blood sampling and urine collections. Each participant will be in this study for up to approximately 4 years. Tovorafenib will be provided to participants who tolerate it for as long as their disease does not progress.\n\nOnce tovorafenib becomes approved and commercially available in Japan, participants may transition to the commercial drug for continued treatment.\n\nA participant may withdraw consent to participate at any time.",[258],"Low-grade Glioma",[260,261],"BRAF-altered Recurrent Low-grade Glioma","Progressive Low-grade Glioma","2026-06-30",{"date":27,"type":30},{"date":265,"type":30},"2026-03-03",{"date":267,"type":20},"2030-07-31",{"name":36,"class":37},{"id":270,"slug":271,"hasResults":11,"nctId":272,"briefTitle":273,"officialTitle":274,"acronym":4,"eligibilityCriteria":275,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":276,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":277,"conditions":278,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":280,"startDateStruct":281,"completionDateStruct":283,"leadSponsor":285,"locationsCount":286},"100617095","study-of-cabozantinib-in-participants-with-neuroendocrine-tumors-who-have-already-received-prior-treatment-100617095","NCT07314164","Study of Cabozantinib in Participants With Neuroendocrine Tumors Who Have Already Received Prior Treatment","CaboLife: A Prospective Non-Interventional Study on Effectiveness and Safety of Cabozantinib in Real-Life Setting for Previously Treated Patients With Neuroendocrine Tumour","Inclusion Criteria:\n\nParticipants can join the study if they meet all of the following conditions:\n\n* Are 18 years or older and able to give informed consent.\n* Have a physician-initiated decision to start treatment with cabozantinib for neuroendocrine tumors (NETs), made before joining the study.\n* Have unresectable or metastatic, well-differentiated pancreatic (pNET) or extra-pancreatic neuroendocrine tumors (epNET).\n* Have already received at least one prior systemic therapy, other than somatostatin analogues.\n* Have signed a written informed consent form.\n\nExclusion Criteria:\n\nParticipants cannot join the study if:\n\n* They are currently participating in an interventional clinical trial, or have done so within the last 3 months before joining this study.\n* They have a contraindication to cabozantinib treatment, based on the product's official prescribing information.",{"count":116,"type":20},"This study will assess how well cabozantinib works and how safe it is in adults with a type of cancer called neuroendocrine tumors (NETs).\n\nThese tumors can appear in all parts of the body. All participants in this study have already received at least one treatment that affects the whole body to help manage their cancer, but their disease has continued to grow.\n\nThe study will take place in regular hospitals and clinics in Germany and Austria.\n\nIt will follow about 150 participants who are taking cabozantinib as part of their usual care. Doctors will collect information from routine medical visits, tests, and scans to see how the cancer responds to treatment and how long participants stay on cabozantinib. They will also look at side effects and how the treatment affects participants' quality of life.\n\nThis is an observational study, which means that no extra tests or procedures will be done beyond what is normally used to care for participants with this condition.",[279],"Neuroendocrine Tumors",{"date":27,"type":30},{"date":282,"type":30},"2025-12-17",{"date":284,"type":20},"2028-12-31",{"name":36,"class":37},34,{"id":288,"slug":289,"hasResults":11,"nctId":290,"briefTitle":291,"officialTitle":292,"acronym":4,"eligibilityCriteria":293,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":294,"targetDuration":4,"studyType":94,"phases":296,"briefSummary":298,"conditions":299,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":302,"startDateStruct":303,"completionDateStruct":305,"leadSponsor":307,"locationsCount":308},"100609379","phase-1-a-study-to-assess-the-safety-tolerability-pharmacokinetic-pharmacodynamic-immunogenicity-and-antitumour-activity-of-ipn60300-in-adults-with-locally-advanced-or-metastatic-solid-tumours-100609379","NCT07213817","A Study to Assess the Safety, Tolerability, Pharmacokinetic, Pharmacodynamic, Immunogenicity and Antitumour Activity of IPN60300 in Adults With Locally Advanced or Metastatic Solid Tumours","An Open-Label, Phase I\u002FII First-in-Human, Dose Escalation, Dose Optimisation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacokinetic, Pharmacodynamic, Immunogenicity and Antitumour Activity of IPN60300 as Single Agent in Adult Participants With Locally Advanced or Metastatic Solid Tumours.","Inclusion criteria:\n\n* Participant must be ≥18 years of age, at the time of signing the informed consent.\n* Participants with histologically or cytologically documented, locally advanced, or metastatic solid tumors, that relapsed or were refractory after being previously treated with standard of care therapy; or for which there is no available established therapy; or standard therapy is contraindicated or not deemed appropriate by the treating investigator.\n* Participants must have measurable disease per Response Evaluation Criteria in Solid Tumours (RECIST) version 1.1.\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.\n* Male and female participants Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Adequate bone marrow function within 7 days before first dose of study intervention,\n* Adequate renal function within 7 days before first dose of study intervention,\n* Adequate hepatic function or laboratory abnormalities indicating hepatic injury within 7 days before first dose of study intervention,\n* Prothrombin time or international normalised ratio (INR) ≤1.5 × ULN.\n* At the time of screening, a tumour tissue specimen is required for enrolment into the dose escalation and dose optimisation portions of the study for retrospective central laboratory determination.\n* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF).\n* Have a life expectancy of more than 3 months for disease-related mortality, as evaluated by the investigator.\n\nExclusion Criteria:\n\n* Known second malignancy either progressing or requiring active treatment within the last 2 years prior to first dose of study intervention.\n* Residual toxicity from prior anticancer therapy that are NCI CTCAE version 5.0 Grade 2 or higher. Stable chronic Grade 2 toxicities from previous treatments may be eligible per the judgement of investigator.\n* History of major surgery within 4 weeks prior to the first dose of study intervention.\n* Previous solid organ transplantation.\n* Pre-existing, acute or chronic severe corneal disorders, sequelae from severe corneal disorders, or a history of corneal transplantation.\n* Active brain metastases or leptomeningeal metastases with exception to asymptomatic and treated brain metastases (i.e. no neurological symptoms, no requirements for corticosteroids and lesions \\\u003C1.5 cm), which are stable and not expected to become symptomatic in the next 3 months in the opinion of the investigator.\n* History of stroke or significant cerebrovascular disease (ie, transient ischemic attack) within 6 months prior to initiation of study intervention.\n* History of clinically significant cardiac disease within 6 months prior to the initiation of study intervention, including but not limited to unstable angina, acute myocardial infarction, endoscopic or open-heart cardiac surgery, or heart failure classified as New York Heart Association Grade 2 or higher.\n* History of clinically significant respiratory disease within 6 months prior to the initiation of study intervention, including severe chronic obstructive pulmonary disease or asthma.\n* History of noninfectious interstitial lung disease (ILD)\u002Fpneumonitis\u002Fradiation pneumonitis that required steroids or has current ILD\u002Fpneumonitis.\n* Clinically significant gastrointestinal disorder including bleeding, occlusion, diarrhoea \\>Grade 1, malabsorption syndrome, ulcerative colitis, inflammatory bowel disease or partial bowel obstruction.\n* Any evidence of severe active infection or inflammatory condition.\n* Significant concurrent, uncontrolled medical condition that would put participants at unacceptable risk from study participation or preclude them from complying with study procedures as per investigator assessment, including, but not limited to renal, hepatic, haematological, gastrointestinal, endocrine, pulmonary, neurological, cerebral, or psychiatric disease.\n* Participants with uncontrolled human immunodeficiency virus (HIV). HIV infected participants are eligible if they meet criteria described in the protocol.\n* Known active infection with hepatitis B virus (HBV) OR hepatitis C virus (HCV). Participants are eligible if they meet criteria described in the protocol.\n* Ongoing immunosuppressive therapy, including systemic corticosteroids. NOTE: Physiologic replacement or use of topical or inhaled corticosteroids are allowed.\n* Concurrent participation in another therapeutic treatment trial, previous participation should respect the minimum of 5 half-lives or 4 weeks before the study intervention initiation (whichever is shorter).\n* Participants accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalised.\n* For French participants only: participants are under court protection, not affiliated to a social security system or protected adults.",{"count":295,"type":20},114,[255,297],"PHASE2","This study aims to find the right dosage and evaluate the safety and effectiveness of the drug IPN60300 in adults with advanced solid tumours, which are cancers that have spread to other parts of the body from their original location. All participants will receive the drug by injection.\n\nStudy Phases:\n\n* Phase Ia: Participants with certain types of tumours will be treated in cohorts of increasingly higher doses of the drug to determine the safe and effective dose range (a high and a low dose).\n* Phase Ib: Participants with a specific tumour type will receive one of the two doses identified in phase Ia. The dose level will be assigned randomly (by chance).\n\nStudy Periods:\n\nScreening: Up to 28 days before first IPN60300 injection to determine eligibility.\n\nTreatment: Starts with the first dose of IPN60300 and continues until it needs to be stopped due to harmful effects, the disease getting worse, or if the participant decides to stop taking part in the study, the investigator's decision to stop treatment, death or the study is terminated early by the sponsor.\n\nParticipants will undergo blood tests, urine collections, physical examinations, and clinical evaluations.",[300,301],"Locally Advanced Solid Tumor","Metastatic Solid Tumor",{"date":27,"type":30},{"date":304,"type":30},"2025-12-05",{"date":306,"type":20},"2028-10-30",{"name":36,"class":37},12,{"id":310,"slug":311,"hasResults":11,"nctId":312,"briefTitle":313,"officialTitle":314,"acronym":4,"eligibilityCriteria":315,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":316,"targetDuration":4,"studyType":94,"phases":318,"briefSummary":319,"conditions":320,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":322,"startDateStruct":323,"completionDateStruct":325,"leadSponsor":327,"locationsCount":328},"100609380","phase-1-a-study-to-assess-the-safety-tolerability-pharmacokinetics-pharmacodynamics-immunogenicity-and-anti-tumour-activity-of-ipn01203-in-adults-with-locally-advanced-or-metastatic-solid-tumours-exposed-to-immune-checkpoint-inhibitor-therapies-100609380","NCT07213830","A Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity and Anti-tumour Activity of IPN01203 in Adults With Locally Advanced or Metastatic Solid Tumours Exposed to Immune Checkpoint Inhibitor Therapies","An Open-label, Phase I\u002FII First in Human, Dose Escalation, Optimisation and Expansion Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity and Anti-tumour Activity of IPN01203 in Participants With Locally Advanced or Metastatic Solid Tumours Who Have Progressed on or After Immune Checkpoint Inhibitor Therapies","Inclusion Criteria:\n\n* Participant must be ≥18 years of age, at the time of signing the informed consent.\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 1.\n* Measurable disease per RECIST version 1.1 (at least one lesion that is measurable by RECIST 1.1. Tumour lesions in a previously irradiated area are considered measurable if progression has been demonstrated in such lesions after radiation) and documented locally advanced or metastatic disease with CT and\u002For MRI.\n* All acute, clinically significant (CS) treatment-related AEs from a prior therapy resolved to Grade 1 or lower prior to study entry. Participants with chronic toxicities such as Grade ≤2 neuropathy or alopecia can be included.\n* Have a life expectancy for disease-related mortality, as evaluated by the investigator.\n* Male and female participants: Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Adequate haematologic and end organ function\n* Participant is capable of giving signed informed consent as described in the protocol.\n\nExclusion Criteria:\n\n* Have untreated or active primary brain tumour, Central Nervous System (CNS) metastases, leptomeningeal disease, or spinal cord compression.\n* Experienced severe, life-threatening immune-mediated AEs, or infusion-related reactions such as those that lead to permanent discontinuation while on treatment with prior anticancer therapy such as immune checkpoint inhibitor therapy.\n* History of known autoimmune disease\n* History of stroke or significant cerebrovascular disease, encephalitis, meningitis, organic brain disease (e,g., Parkinson's disease) or uncontrolled seizures in the year prior to first dose of study drug.\n* History of CS cardiac disease within 6 months prior to the initiation of study intervention, including but not limited to unstable angina, acute myocardial infarction, endoscopic or open-heart cardiac surgery, or heart failure classified as New York Heart Association Grade 2 or higher. Additional exclusion criteria include:\n\n  1. Left ventricular ejection fraction \\\u003C45%\n  2. QT interval corrected by Fridericia (QTcF) \\>470 ms (for women) and \\>450 ms (for men) or CS arrhythmias.\n* History of CS respiratory disease within 6 months prior to the initiation of study intervention, including severe chronic obstructive pulmonary disease or asthma.\n* Prior organ transplantation.\n* Chronic or ongoing active infections within 4 weeks prior to Cycle1 Day1 (C1D1).\n* Presence of hepatitis B surface antigen (HBsAg) \\[or hepatitis B core antibody (HBcAb)\\] at screening or within 3 months prior to the first dose of study intervention.\n* Positive hepatitis C antibody test result at screening or within 3 months prior to the first dose of study intervention.\n* Participants with known history of HIV infection are excluded from the study unless they meet the following criteria:\n\n  1. Stable Antiretroviral Therapy: Participants must be on a stable antiretroviral therapy regimen for at least 4 weeks prior to enrolment.\n  2. CD4+ T cell Count: Participants must have a CD4+ T cell count of at least 200 cells\u002FµL.\n  3. Viral Load: Participants must have an undetectable viral load (HIV RNA \\\u003C50 copies\u002FmL)\n  4. No Opportunistic Infections: Participants must not have had any opportunistic infections or other human immunodeficiency virus (HIV)-related illness within the past 6 months Note: HIV testing will be performed in any countries where it is mandatory per local requirements.\n* History of other malignancy within the last years.\n* Significant concurrent, uncontrolled medical condition that would put participants at unacceptable risk from study participation or preclude them from complying with study procedures per investigator including, but not limited to renal, hepatic, haematologic, gastrointestinal, endocrine, pulmonary, neurological, cerebral, or psychiatric disease.\n* Treatment with \\>10 mg per day of prednisone (or equivalent) or other immune suppressive drugs within 7 days prior to the initiation of study drug. Exceptions may be made for participants who have had allergic reaction to iodinated contrast media. Steroids for topical, ophthalmic, inhaled, or nasal administration are allowed.\n* Concurrent participation in another therapeutic treatment study.\n* Participants accommodated in an institution because of regulatory or legal order; prisoners or participants who are legally institutionalised.\n* For French participants only: participants are under court protection, not affiliated to a social security system or protected adults.",{"count":317,"type":20},102,[255,297],"The purpose of this study is to determine the appropriate dosage, safety and effectiveness of a new drug, IPN01203, in adults with advanced solid tumours.\n\nAdvanced solid tumours are cancers that can occur in various organs or tissues and have spread from their original site to nearby tissues or other parts of the body.\n\nThere will be two parts to this study:\n\n* Phase Ia: This part (called dose escalation) will find the dose range that shows activity against the tumour and can be tolerated by participants by testing different increasing doses of IPN01203.\n* Phase Ib: This part (called dose optimisation) will assess the ability of the drug to prevent, slow down, or stop the growth of tumours and how the body processes and responds to the drug when given in \"low dose\" or \"high dose.\" It will also further explore the safety and tolerability.\n\nAn additional part (phase II) may be added to the study based on the results of phase Ia and phase Ib.\n\nEach part will consist of the following periods:\n\n* A screening period (up to 28 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre.\n* A treatment period where all eligible participants will receive IPN01203. Requires approximately 15 visits for the first 2 months followed by 3 visits every month from month 3 until unacceptable toxicity, disease progression, death, upon participant's withdrawal of consent, investigator decision, or study termination by the sponsor, whichever occurs first.\n\nThere will also be one visit at the end of treatment (EoT), 30 days after the last administration of the study intervention or prior to the start of new anticancer treatment, whichever is earlier. Additionally, there will be one visit (the safety follow-up visit) 90 days after the last administration of study intervention or prior to the start of new anticancer treatment, whichever is earlier.\n\nIn both parts of the study, participants will undergo blood sampling, urine collection, physical examinations and clinical evaluations. They may continue some other medications, but the details need to be recorded.\n\nEach participant will be in this study until death or withdrawal from the study. IPN01203 will be provided to participants who tolerate it for as long as their disease does not progress. Participants may withdraw consent to participate at any time.",[321,301],"Advanced Solid Tumor",{"date":27,"type":30},{"date":324,"type":30},"2026-02-06",{"date":326,"type":20},"2032-07-14",{"name":36,"class":37},11,{"id":330,"slug":331,"hasResults":11,"nctId":332,"briefTitle":333,"officialTitle":334,"acronym":4,"eligibilityCriteria":335,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":336,"targetDuration":4,"studyType":94,"phases":338,"briefSummary":339,"conditions":340,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":341,"startDateStruct":342,"completionDateStruct":344,"leadSponsor":346,"locationsCount":347},"100580108","phase-1-a-study-to-assess-a-new-medicine-called-ipn01195-when-administered-alone-in-adults-with-advanced-solid-tumours-100580108","NCT06833008","A Study to Assess a New Medicine Called IPN01195 When Administered Alone in Adults With Advanced Solid Tumours","An Open-label, Phase I\u002FII First-in-human, Dose Escalation and Confirmation Study to Evaluate the Safety, Tolerability, Pharmacokinetic, Pharmacodynamic and Anti-tumour Activity of IPN01195 as Single Agent in Adult Participants With Advanced Solid Tumours","Inclusion Criteria:\n\n* Participants must be ≥18 years of age or the country's legal age of majority if the legal age is more than 18 years at the time of signing the informed consent.\n* Participants with histologically confirmed metastatic solid tumour for whom no suitable alternative standard therapy exists.\n* Participants must bear tumours harbouring selected classes of genetic alterations of MAPK pathway based on an analytically validated assay performed by an accredited laboratory.\n* Part A: Participants must consent to the use of archival tumour tissue or, if not available, collection of fresh tumour biopsy at screening, for central confirmation of mutation status.\n* Part B: Participants must consent to the use of archival tumour tissue or, if not available, collection of fresh tumour biopsy at screening, for MAPK genomic testing to confirm eligibility.\n* Participants must have measurable disease per Response Evaluation Criteria in Solid Tumours (RECIST) version 1.1\n* Eastern Cooperative Oncology Group (ECOG)\u002Fperformance status (PS) of 0 or 1\n* Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.\n\nExclusion Criteria:\n\n* Gastrointestinal conditions that could impair absorption of IPN01195 (specific cases e.g. remote history of gastrointestinal surgery, may be enrolled after discussion with the medical monitor)\n* Any evidence of severe active infection or inflammatory condition.\n* Non-adequate cardiac function\n* Known psychiatric or substance abuse disorder, or any other cognitive disorder per the opinion of the investigator that would interfere with the participant's ability to cooperate with the requirements of the study.\n* Underlying medical conditions that, in the investigator's or sponsor's opinion, will obscure the interpretation of toxicity determination or AEs.\n* Known second malignancy either progressing or requiring active treatment within the last 2 years prior to first dose of the study intervention.\n* Active brain metastases or leptomeningeal\n* Current enrolment or past participation in any other clinical studies involving an investigational study treatment within the last 28 days\n* Live vaccine(s) within 28 days prior to first dose of the study intervention or plan to receive such vaccines during the study.\n* Concurrent treatment with any other anti-cancer therapy (including radiotherapy or investigational agents).\n* Washout period of less than 28 days prior anti-cancer therapy (including chemotherapy, targeted agents, radiotherapy). If the participant was treated with an agent having a short half-life, washout can be \\\u003C28 days but not shorter than 5 times the half-life.\n* Condition requiring systemic treatment with either corticosteroids (\\>10 mg daily prednisone equivalents) or other immunosuppressive medications within 2 weeks prior to first dose of the study intervention.\n* Non-adequate bone marrow function\n* Non-adequate renal function\n* Non-adequate hepatic function\n* Known human immunodeficiency virus (HIV) infection. HIV testing will be performed in any countries where mandatory per local requirements.\n* Known uncontrolled or untreated hepatitis infection.\n\n  * (a) Known uncontrolled hepatitis B virus (HBV) infection.\n  * (b) Known untreated current hepatitis C virus (HCV) infection.\n* Sensitivity to IPN01195 or any of its components.",{"count":337,"type":20},85,[255,297],"The purpose of this study is to determine the appropriate dosage, safety and effectiveness of a new study drug IPN01195 in adults with advanced solid tumours.\n\nThe participants in this study will have advanced solid tumours. 'Advanced solid tumours' refers to cancers that can occur in several places, including cancers in organs or tissues that have spread from their original site to nearby tissues or other parts of the body.",[321],{"date":27,"type":30},{"date":343,"type":30},"2025-03-14",{"date":345,"type":20},"2028-10-03",{"name":36,"class":37},13,{"id":349,"slug":350,"hasResults":11,"nctId":351,"briefTitle":352,"officialTitle":353,"acronym":354,"eligibilityCriteria":355,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":4,"enrollmentInfo":356,"targetDuration":4,"studyType":94,"phases":358,"briefSummary":359,"conditions":360,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":363,"lastUpdatePostDateStruct":364,"startDateStruct":366,"completionDateStruct":368,"leadSponsor":369,"locationsCount":4},"100640433","phase-2-a-study-to-assess-how-well-the-study-medicine-ipn60340-works-in-combination-with-azacitidine-and-venetoclax-compared-to-placebo-in-combination-with-azacitidine-and-venetoclax-in-participants-with-newly-diagnosed-acute-myeloid-leukemia-who-cannot-receive-intensive-chemotherapy-100640433","NCT07623187","A Study to Assess How Well the Study Medicine IPN60340 Works in Combination With Azacitidine and Venetoclax, Compared to Placebo in Combination With Azacitidine and Venetoclax, in Participants With Newly Diagnosed Acute Myeloid Leukemia Who Cannot Receive Intensive Chemotherapy","A Two-part, Phase 2b\u002FPhase 3 Double-blinded, Randomized Study of IPN60340 in Combination With Azacitidine and Venetoclax Versus Placebo in Combination With Azacitidine and Venetoclax in Participants With Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy.","EVICTION 3","Inclusion Criteria:\n\n1. Participant must be 18 years of age or older, at the time of signing the informed consent.\n2. Have newly diagnosed AML, as per WHO 2022 criteria.\n3. Eastern Cooperative Oncology Group (ECOG) performance status of 1 to 2 for participants ≥75 years of age, or 1 to 3 for participants \\\u003C75 years of age\n4. Participants must be considered ineligible for intensive chemotherapy, due to age or comorbidities,\n5. Adequate organ function as indicated in the protocol\n6. Contraceptive use by participant or participant partners should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials.\n7. Signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol\n\nExclusion Criteria:\n\nParticipants are excluded from the study if any of the following criteria apply:\n\n1. Current diagnosis of:\n\n   I. Acute promyelocytic leukemia (APL) II. Active or uncontrolled central nervous system (CNS) leukemia III. Any γ9δ2TC neoplasm\n2. History of myeloproliferative neoplasms (MPN) including primary myelofibrosis, essential thrombocythemia, polycythemia vera, chronic myeloid leukemia, or MDS\u002FMPN as per WHO 2022 or treatment-related AML\n3. History of other malignancy within the last 2 years.\n4. Rapidly progressing disease in the opinion of the clinical investigator which may preclude treatment in this study.\n5. History of clinically significant or uncontrolled cardiac disorders, within 6 months prior to Cycle 1 Day 1 (C1D1)\n6. White blood cell (WBC) count \\>25 × 10\\^9\u002FL . Cytoreduction can be used before C1D1 and beyond as needed to keep WBC \\\u003C 25 × 10\\^9.\n7. Participants with severe hepatic impairment, e.g., Child-Pugh C, are excluded.\n8. Major surgery within 4 weeks prior to C1D1 or planned during the foreseeable duration of the study.\n9. Any gastrointestinal disorder or malabsorption syndrome that may impair absorption of venetoclax\n10. Uncontrolled or severe bacterial, fungal, viral, and\u002For parasitic infections treated with therapeutic oral or intravenous anti-infective agents. Prophylactic antimicrobials are allowed.\n11. Uncontrolled human immunodeficiency virus (HIV) disease will be excluded. Participants on anti-retroviral therapy should be included as long as their disease is under control, taking precautions to modify their highly active antiretroviral therapy (HAART) regimen to minimize drug interactions.\n12. Presence of hepatitis B surface antigen (HBsAg) and\u002For hepatitis B core antibody (HBcAb) at screening or within 3 months prior to randomization.\n\n    NOTE: Participants with known positive HBsAb may be randomized provided they are hepatitis B-vaccinated and have negative HBsAg and HBcAb.\n13. Positive hepatitis C antibody test result at screening or within 3 months of randomization unless HCV-RNA negative test is documented.\n\n    NOTE: Participants with positive hepatitis C antibody due to prior resolved disease can be enrolled if a confirmatory negative hepatitis C ribonucleic acid (RNA) test is obtained.\n14. Participant has received strong and\u002For moderate cytochrome P450 (CYP)3A inducers within 7 days prior to the initiation of study treatment.\n15. Participant is unable to swallow capsules or tablets\n16. Prior treatment with hypomethylating agents, chemotherapy, B-cell lymphoma protein (BCL) 2 inhibitors, clinical trial therapy, cellular therapy or allogenic hematopoietic cell transplantation (HCT) for MDS.\n17. Treatment with systemic corticosteroids of \\>10 mg\u002Fday prednisone (or equivalent) or other systemic immunosuppressive medications within 5 half-lives prior to C1D1, or anticipated requirement for systemic immunosuppressive medications during the study.\n18. Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the investigator \\[or medical monitor\\], contraindicates participation in the study.",{"count":357,"type":20},450,[297,96],"The purpose of this study is to find out how well the study drug IPN60340 works to treat participants with acute myeloid leukemia. Acute myeloid leukemia is a rare blood cancer that grows quickly. This study's main aim is to compare the percentage of participants who reach complete remission within the first 6 months of treatment between the 2 study arms (study drug and standard medicines compared to placebo and standard medicines).\n\nIn this study all participants will receive azacitidine and venetoclax plus either the study drug IPN60340 or placebo. Venetoclax will be given as a tablet by mouth once each day in 28-day cycles. Azacitidine will be given by injection under the skin (subcutaneously) or through the veins (intravenously) daily for the first 7 days of each 28-day cycle. IPN60340 or placebo (depending on which arm of the study the participant is assigned to) will be given through the veins (intravenously) on day 1 of each 28-day cycle.\n\nThere will be 4 periods in this study:\n\n* A screening period (up to 28 days) to assess whether the participant can take part requiring at least 1 visit to the study center.\n* A treatment period where all eligible participants will receive azacitidine and venetoclax plus either the study drug IPN60340 or placebo. The study requires 8 visits for the first month followed by 1 visit every month until unacceptable toxicity, disease progression, the start of new cancer treatment, or study closure, whichever is first.\n* A safety follow-up period (at 28 days (±3 days) after the last dose of study medicine) to assess safety after participants have finished treatment.\n* A long-term follow-up period where participants' health will be monitored using a telephone call or clinic visit every 12 weeks until the end of study.\n\nParticipants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), bone marrow aspirates (sampling of the liquid part of the bone marrow). Some participants will also undergo pregnancy testing. Participants in the Phase 3 portion of the study will also be asked to fill in questionnaires.\n\nThe time each participant will be in this study will vary based on how well the medicine works to treat the participant's AML. Azacitidine and venetoclax plus either IPN60340 or placebo will be provided to participants who tolerate it for as long as their disease does not progress. Participants may withdraw consent to participate at any time.",[361,362],"Acute Myeloid Leukaemia (AML)","Acute Myeloid Leukaemia","2026-05-29",{"date":365,"type":30},"2026-06-03",{"date":367,"type":20},"2026-11-01",{"date":199,"type":20},{"name":36,"class":37},{"id":371,"slug":372,"hasResults":11,"nctId":373,"briefTitle":374,"officialTitle":375,"acronym":376,"eligibilityCriteria":377,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":378,"enrollmentInfo":379,"targetDuration":4,"studyType":94,"phases":381,"briefSummary":382,"conditions":383,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":385,"lastUpdatePostDateStruct":386,"startDateStruct":388,"completionDateStruct":390,"leadSponsor":392,"locationsCount":393},"100625835","phase-3-a-study-to-assess-the-effectiveness-and-safety-of-ipn10200-in-adults-with-moderate-to-severe-wrinkle-like-lines-between-the-eyebrows-100625835","NCT07427797","A Study to Assess the Effectiveness and Safety of IPN10200 in Adults With Moderate to Severe Wrinkle-like Lines Between the Eyebrows","A Phase III, Multicentre, Double-blind, Randomised, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of a Single Dose of IPN10200 in the Improvement of Moderate to Severe Glabellar Lines in Adult Participants","LAURITE 1","Inclusion Criteria:\n\n* Participant should be male or female, ≥18 years of age inclusive at the time of signing the ICF.\n* Moderate or severe (Grade 2 or 3) GL at MF at baseline, as assessed by the ILA using a validated 4-point photographic scale.\n* Moderate or severe (Grade 2 or 3) GL at MF at baseline, as assessed by the SSA using a 4-point categorical scale.\n* Dissatisfied or very dissatisfied (Grade 2 or 3) with their lines at baseline, as assessed by the SLS.\n* For female participants: Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n* Participant has both the time and the ability to complete the study and comply with study instructions.\n* Does not reside in an institution by administrative or court order.\n* Is not a sponsor employee or clinical research unit personnel directly affiliated with the study or is not an immediate family member. Immediate family is defined as a spouse, parent, child or sibling whether biological or legally adopted.\n\nExclusion Criteria:\n\n* An active infection or other skin problems in the upper face including the GL area (e.g. acute acne lesions or ulcers).\n* A history of eyelid blepharoplasty or brow lift or any other upper facial surgery within the past 5 years.\n* A history of facial nerve palsy.\n* Marked facial asymmetry, ptosis, excessive dermatochalasis, deep dermal scarring or thick sebaceous skin.\n* Presence of any scars, piercings or tattoos (including micro blading of the eyebrows) in or around the treatment area that have occurred within 6 months prior to baseline, or which in the investigator's opinion, could interfere with evaluations.\n* Any known medical condition that may put the participant at increased risk in regard to exposure to BoNT of any serotype (i.e. myasthenia gravis, Eaton-Lambert syndrome, amyotrophic lateral sclerosis, etc.).\n* Administration of any BoNT (other than the study intervention on baseline visit) into any site of the body and for any indication from 9 months prior to the first study visit until the end of the study.\n* Participants who have received IPN10200 in a previous study.\n* Use of medications that affect neuromuscular transmission (such as curare-like nondepolarising agents, lincosamides, polymyxins, anticholinesterases) within the past 30 days prior to baseline are prohibited or a longer washout period of at least five half-lives might be required, as deemed appropriate by the investigator for long-acting medications.\n* Use of aminoglycoside antibiotics within the past 30 days prior to baseline are prohibited. Note: Topical use apart from the area of injection would be acceptable.\n* Use of systemic retinoids within the past 30 days prior to baseline and planned use during the study. Note: Topical retinoids are allowed other than in the areas that will be injected (upper facial area) at the discretion of the investigator.\n* Any prior treatment with permanent fillers, lifting threads, autologous fat or permanent procedures in the upper face including the GL area.\n* Administration of any non-permanent injectables (such as hyaluronic acid, calcium hydroxylapatite, poly-L-Lactic acid or polymethyl-methacrylate) soft tissue augmentation therapy in the GL region within 12 months prior to baseline.\n* Any prior facial treatment or aesthetic procedures to the upper face including photo rejuvenation, vascular or pigment laser or microneedling within the 3 months prior to baseline.\n* Any prior facial treatment or aesthetic procedures to the upper face involving skin resurfacing (including dermabrasion, laser or whatever the interventional technique used) or chemical peel within the past 12 months prior to baseline.\n* Any planned cosmetic surgery or aesthetic procedures to the upper face during the study and\u002For any procedures to other parts of the face which in the investigator's opinion, could interfere with evaluations during the study.\n* Any past surgery in the UFL area including GL.\n* Planned use of concomitant therapy which, in the investigator's opinion, would interfere with the evaluation of the safety or efficacy of the study intervention. Therapy considered necessary for the participant's welfare may be given at the discretion of the investigator. Note: If the permissibility of a specific medication\u002Ftreatment is in question, the medical monitor will be contacted.\n* Use of any experimental device within 30 days prior to baseline visit or use of any treatment with an experimental drug within five times the documented terminal half-life of the respective drug or its metabolites or if the half-life is unknown within 30 days prior to the start of the study (prior to baseline visit) and during the conduct of the study.\n* Known positive for hepatitis B antigen, hepatitis C virus antibody or for human immunodeficiency virus or a diagnosis of acquired immunodeficiency syndrome.\n* Clinically diagnosed significant anxiety disorder, or any other significant psychiatric disorder (e.g. depression) that might interfere with the participant's participation in the study.\n* An inability to substantially lessen GL as determined by the investigator.\n* Known allergy or hypersensitivity to BoNT or any excipients of IPN10200.\n* A history of chronic or recreational drug abuse as assessed by the investigator.\n* Any uncontrolled systemic disease or other significant medical condition which would be harmful for the participant to be entered into the study or continue participation.","80 Years",{"count":380,"type":20},300,[96],"The purpose of this study is to assess the effectiveness and safety of IPN10200 compared to placebo in the improvement of the appearance of moderate to severe glabellar lines in adult participants. Glabellar lines are wrinkle-like lines that appear between the eyebrows and can become more noticeable with age or repeated facial expressions. They may affect a person's appearance and confidence.\n\nIn this study, all participants will receive a single injection of either IPN10200 or placebo (the study drug). There will be 3 periods in this study:\n\n* A screening period (up to 20 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre.\n* A treatment period (Day 1) where all the eligible participants will receive a single injection of IPN10200 or placebo.\n* A follow-up period (52 weeks) where participants' health will be monitored, consisting of regular visits to the study centre and 1 telephone call visit.\n\nParticipants will undergo health measurements and observation, including blood sampling, physical examinations, clinical evaluations and electrocardiograms (ECG: recording of the electrical activity of heart). They will also be asked to fill in questionnaires and keep a diary. Each participant will be in this study for up to 55 weeks. Participants may withdraw consent to participate at any time.",[384],"Moderate to Severe Glabellar Lines","2026-04-29",{"date":387,"type":30},"2026-04-30",{"date":389,"type":30},"2026-02-27",{"date":391,"type":20},"2027-07-15",{"name":36,"class":37},23,{"id":395,"slug":396,"hasResults":11,"nctId":397,"briefTitle":398,"officialTitle":399,"acronym":400,"eligibilityCriteria":401,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":378,"enrollmentInfo":402,"targetDuration":4,"studyType":94,"phases":404,"briefSummary":405,"conditions":406,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":385,"lastUpdatePostDateStruct":407,"startDateStruct":408,"completionDateStruct":410,"leadSponsor":412,"locationsCount":413},"100626422","phase-3-a-study-to-assess-the-effectiveness-and-safety-of-ipn10200-over-time-in-adults-with-moderate-to-severe-wrinkle-like-lines-between-the-eyebrows-100626422","NCT07435428","A Study to Assess the Effectiveness and Safety of IPN10200 Over Time in Adults With Moderate to Severe Wrinkle-like Lines Between the Eyebrows","A Phase III, Multicentre, Double-blind, Randomised, Placebo-controlled and Open-label Study to Evaluate the Efficacy and Safety of a Single Dose of IPN10200 in the Improvement of Moderate to Severe Glabellar Lines in Adult Participants, and to Evaluate the Long-term Efficacy and Safety of Repeat Doses of IPN10200 in the Same Indication","LAURITE 2","Inclusion Criteria:\n\n* Participant should be male or female, ≥18 years of age at the time of signing the informed consent.\n* Moderate or severe (Grade 2 or 3) GL at MF at baseline, as assessed by the ILA using a validated 4-point photographic scale.\n* Moderate or severe (Grade 2 or 3) GL at MF at baseline, as assessed by the SSA using a 4-point categorical scale.\n* Are 'dissatisfied' or 'very dissatisfied' with their GLs at baseline, as assessed by the SLS score.\n* For female participants: Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n* Participant has both the time and ability to complete the study and comply with study instructions.\n* Does not reside in an institution by administrative or court order.\n* Is not a sponsor employee or clinical research unit personnel directly affiliated with the study or is not an immediate family member. Immediate family is defined as a spouse, parent, child or sibling whether biological or legally adopted.\n\nExclusion Criteria:\n\n* An active infection or other skin problems in the upper face including the GL area (e.g. acute acne lesions or ulcers).\n* A history of eyelid blepharoplasty or brow lift or any other upper facial surgery within the past 5 years.\n* A history of facial nerve palsy.\n* Marked facial asymmetry, ptosis, excessive dermatochalasis, deep dermal scarring or thick sebaceous skin.\n* Closed-angle glaucoma or a predisposition to it (for Japan only).\n* Any known medical condition that may put the participant at increased risk with regard to exposure to BoNT of any serotype (i.e. myasthenia gravis, Eaton-Lambert syndrome, amyotrophic lateral sclerosis, etc.).\n* Presence of any scars, piercings, or tattoos (including microblading of the eyebrows) in or around the treatment area that have occurred within 6 months prior to baseline, or which in the investigator's opinion, could interfere with evaluations.\n* Administration of any BoNT (other than the study intervention) into any site of the body and for any indication from 9 months prior to the first study visit until the end of the study.\n* Treatment with IPN10200 in any prior study.\n* Use of medications that affect neuromuscular transmission (such as curare-like nondepolarising agents, lincosamides, polymyxins, anticholinesterases) within the past 30 days prior to baseline is prohibited or a longer washout period of at least five half lives might be required, as deemed appropriate by the investigator for long-acting medications.\n* Use of aminoglycoside antibiotics within the past 30 days prior to baseline are prohibited. Note: Topical use apart from the area of injection would be acceptable.\n* Use of systemic retinoids within the past 30 days prior to baseline and planned use during the study. Note: Topical retinoids are allowed other than in the areas that will be injected (upper facial area) at the discretion of the investigator.\n* Any prior treatment with permanent fillers, lifting threads, autologous fat or permanent procedures in the upper face including the GL area.\n* Administration of any nonpermanent injectables (such as hyaluronic acid, calcium hydroxylapatite, poly-L-lactic acid or polymethyl-methacrylate) for soft tissue augmentation therapy in the GL region within 12 months prior to baseline.\n* Any prior facial treatment or aesthetic procedures to the upper face including photorejuvenation, vascular or pigment laser or microneedling within the 3 months prior to baseline.\n* Any prior facial treatment or aesthetic procedures to the upper face involving skin resurfacing (including dermabrasion, laser, or whatever the interventional technique used) or chemical peel within the past 12 months prior to baseline.\n* Any planned cosmetic surgery or aesthetic procedures to the upper face during the study and\u002For any procedures to other parts of the face which in the investigator's opinion, could interfere with evaluations during the study.\n* Any past surgery in the upper facial line area including GL.\n* Planned use of concomitant therapy which, in the investigator's opinion, would interfere with the evaluation of the safety or efficacy of the study intervention. Therapy considered necessary for the participant's welfare may be given at the discretion of the investigator. Note: If the permissibility of a specific medication\u002Ftreatment is in question, the medical monitor will be contacted.\n* Use of any experimental device within 30 days or use of any treatment with an experimental drug within five times the documented terminal half-life of the respective drug or its metabolites or if the half-life is unknown within 30 days prior to the start of the study (prior to baseline) and during the conduct of the study.\n* Known positive for hepatitis B antigen, or hepatitis C virus antibody, or for human immunodeficiency virus or a diagnosis of acquired immunodeficiency syndrome.\n* Clinically diagnosed significant anxiety disorder, or any other significant psychiatric disorder (e.g. depression) that might interfere with the participant's participation in the study.\n* An inability to substantially lessen GL as determined by the investigator.\n* Known allergy or hypersensitivity to BoNT or any excipients of IPN10200.\n* A history of chronic or recreational drug abuse as assessed by the investigator.\n* Any uncontrolled systemic disease or other significant medical condition which would be harmful for the participant to be entered into the study or continue participation.",{"count":403,"type":20},1300,[96],"The purpose of this study is to assess the effectiveness and safety of a single dose of IPN10200 compared to placebo (double-blind phase) and how well and safely repeat doses of IPN10200 work over time (open-label phase) in adult participants with moderate to severe glabellar lines. Glabellar lines are wrinkle-like lines that appear between the eyebrows and can become more noticeable with age or repeated facial expressions. They may affect a person's appearance and confidence.\n\nAll participants in the double-blind phase will receive IPN10200 or placebo during the first treatment cycle. De novo participants in the open-label phase will receive IPN10200 during the first treatment cycle. Some participants may receive additional treatment cycles with IPN10200 depending on their eligibility.\n\nThere will be 3 periods in this study:\n\n* A screening period (up to 20 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre.\n* A treatment period where participants may receive up to 4 treatment cycles. In the double-blind phase, participants receive a single treatment of IPN10200 or placebo. In the open-label phase (rollover participants from double-blind), eligible participants may receive additional cycles of IPN10200. In the open-label phase (de novo participants), participants will receive IPN10200 in the first cycle and eligible participants may receive additional cycles of IPN10200. Requires multiple visits during the first month followed by 1 visit every month.\n* A follow-up period (24 weeks) after the last injection where participants' health will be monitored.\n\nParticipants will undergo health measurements and observation, including blood sampling, physical examinations, clinical evaluations and electrocardiograms (ECG: recording of the electrical activity of heart). They will also be asked to fill in questionnaires and keep a diary.\n\nEach participant will be in this study for up to 107 weeks. Participants may withdraw consent to participate at any time.",[384],{"date":387,"type":30},{"date":409,"type":30},"2026-02-18",{"date":411,"type":20},"2028-10-01",{"name":36,"class":37},60,{"id":415,"slug":416,"hasResults":11,"nctId":417,"briefTitle":418,"officialTitle":419,"acronym":420,"eligibilityCriteria":421,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":378,"enrollmentInfo":422,"targetDuration":4,"studyType":94,"phases":424,"briefSummary":425,"conditions":426,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":385,"lastUpdatePostDateStruct":428,"startDateStruct":430,"completionDateStruct":432,"leadSponsor":434,"locationsCount":435},"100588172","phase-2-a-study-of-ipn10200-for-the-treatment-of-cervical-dystonia-in-adults-100588172","NCT06937931","A Study of IPN10200 for the Treatment of Cervical Dystonia in Adults","A Phase II, Multicentre, Randomised, Double-blind, Parallel-Group, Placebo Controlled Study to Evaluate the Efficacy and Safety of IPN10200 as a Treatment for Cervical Dystonia in Adult Participants","CATALPA","Inclusion Criteria:\n\n1. A clinical diagnosis of isolated Cervical Dystonia (CD) (idiopathic) characterized by dystonic symptoms localised to the head, neck, and shoulder areas with at least moderate severity at Screening and Baseline (Day 1) defined as:\n\n   * (a) Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total score ≥20\n   * (b) TWSTRS-Severity subscale score ≥15\n   * (c) TWSTRS-Disability subscale score ≥3\n   * (d) TWSTRS-Pain subscale score ≥ 1\n2. Treatment naïve or non-naïve to BoNT therapy for CD\n\nExclusion Criteria:\n\n1. Participants presenting with a swallowing disorder of any origin which might be exacerbated by BoNT treatment, such as:\n\n   * (a) Grade 3 or 4 on the Dysphagia Severity Scale (severe dysphagia) with swallowing difficulties and requiring a change in diet.\n2. Predominant anterocollis.\n3. Predominant retrocollis.\n4. Traumatic torticollis or tardive torticollis.\n5. Marked limitation on passive range of motion that suggests cervical contractures or structural abnormality.",{"count":423,"type":20},132,[297],"The purpose of this study is to evaluate the efficacy and safety of the study drug, IPN10200, and to assess how well it works when compared with placebo in treating Cervical Dystonia (CD) in adults.\n\nCD can cause a series of abnormalities and symptoms in the head and neck that can lead to neck pain and stiffness, and headaches. CD is believed to involve deep parts within the brain that control movement, but genetic factors, environmental factors, and abnormalities in the brain may also play a role.\n\nThe usual treatment for CD includes injecting BoNT into the affected muscles, but the treatment only lasts about 3 months. IPN10200 is designed to last for a longer period.\n\nThe study will consist of two periods:\n\n1. A Screening Period of up to 4 weeks (28 days) to assess whether a participant can take part in the study and requires at least one visit.\n2. A Treatment Period of 36 weeks.\n\nOn Day 1 of the treatment period, participants will receive either IPN10200 Dose A or Dose B (additional participants may receive IPN10200 Dose C) of the study drug, or placebo distributed into different muscles in the head, neck and shoulders. Participants may continue some other medications, but details need to be recorded.\n\nThere will be 10 visits to the clinic in person and one remote visits (phone call) (12 visits to the clinic for participants who receive Dose C). Participants will undergo blood samplings, urine collections, physical\u002Fneurological examinations, and clinical evaluations. Participants will also need to complete questionnaires throughout the study.\n\nThe total study duration for a participant will be up to 40 weeks (approximately 9 months).",[427],"Cervical Dystonia",{"date":429,"type":30},"2026-05-05",{"date":431,"type":30},"2025-06-30",{"date":433,"type":20},"2027-11-05",{"name":36,"class":37},44,{"id":437,"slug":438,"hasResults":11,"nctId":439,"briefTitle":440,"officialTitle":441,"acronym":442,"eligibilityCriteria":443,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":378,"enrollmentInfo":444,"targetDuration":4,"studyType":94,"phases":446,"briefSummary":447,"conditions":448,"keywords":4,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":385,"lastUpdatePostDateStruct":451,"startDateStruct":452,"completionDateStruct":454,"leadSponsor":456,"locationsCount":457},"100564122","phase-2-a-study-to-evaluate-ipn10200-safety-and-efficacy-in-the-prevention-of-episodic-or-chronic-migraine-in-adults-100564122","NCT06625060","A Study to Evaluate IPN10200 Safety and Efficacy in the Prevention of Episodic or Chronic Migraine in Adults","A Multicentre, Randomised, Double-blind, Placebo-controlled, Dose Escalation and Dose Finding Phase II Study to Evaluate the Safety and Efficacy of IPN10200 in the Prevention of Episodic or Chronic Migraine in Adults","MERANTI","Inclusion Criteria:\n\n1. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF. Participant has provided written informed consent and signed privacy\u002Fdata protection documentation;\n2. Male or female ≥18 to 80 years of age at the time of signing the informed consent;\n3. Diagnosis of either EM or CM, per ICHD-3 criteria, for at least 12 months prior to the screening visit;\n4. Diagnosis of migraine at ≤50 years of age;\n5. Participants in the EM group: History of EM diagnosis and headache frequency (i.e. migraine and non-migraine headache): ≤14 headache days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary; migraine frequency: ≥6 migraine days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary;\n6. Participants in the CM group: History of CM diagnosis and headache frequency (i.e. migraine and non-migraine headache): ≥15 headache days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary; migraine frequency: ≥8 migraine days in the 4 weeks prior to randomisation on study Day 1 based on information recorded in the eDiary;\n7. Participant with a history of use of at least one preventive treatment for migraine.\n\nExclusion Criteria:\n\n1. History or current diagnosis of migraine with brainstem aura, retinal migraine, complications of migraine, tension-type headache, trigeminal autonomic cephalalgias, hypnic headache, hemicrania continua or new daily persistent headache;\n2. Headache attributed to another disorder (e.g. secondary headaches), except medication overuse headache (MOH);\n3. Current uncontrolled psychiatric or psychological condition, or one that could confound assessment of headaches\u002Fmigraines or interfere with study participation;\n4. Risk of self-harm or harm to others as evidenced by past suicidal behaviour or endorsing items 3, 4, or 5 on the C-SSRS at screening or Day 1.\n5. Participants presenting with a swallowing disorder of any origin which might be exacerbated by botulinum toxin treatment, such as:\n\n   \\- Grade 3 or 4 on the Dysphagia Severity Scale (severe dysphagia) with swallowing difficulties and requiring a change in diet.\n6. Clinically relevant skin condition or infection that could interfere with injection of study intervention;\n7. Participant has any medical condition or situation that would make them unsuitable for participation in the study;\n8. Participant receiving more than one allowable concomitant migraine preventive treatment;\n9. Known history of an inadequate response to \\>4 medications prescribed for the prevention of migraine (2 of which have different mechanisms of action to botulinum toxin);\n10. Use of any of the following medications in the specified timeframe prior to the screening visit:\n\n    * Botulinum toxin for migraine within 24 weeks (or for any other medical\u002Faesthetic reason within 16 weeks);\n    * Prior use of mAbs blocking CGRP pathway within 12 weeks for preventative treatment of migraine\n    * Prior use of oral CGRP receptor antagonist (gepants) for preventative treatment of migraine within 2 weeks;\n    * Anaesthetic or steroid injection in any region targeted for treatment with study medication within 4 weeks;\n    * Use of cannabidiol or other types of cannabinoids within 30 days;\n    * Use of medical device to treat migraine within 4 weeks (e.g. non-invasive neuromodulation therapies such as nerve stimulation (gammaCore), transcranial magnetic stimulation (cephaly), external trigeminal nerve stimulation, transcutaneous electrical nerve stimulation and peripheral neuroelectrical stimulation);\n    * Use of other intervention to treat migraine that is assessed to interfere with study evaluations within 4 weeks (e.g. acupuncture in the head and neck region, cranial traction, nociceptive trigeminal inhibition, occipital nerve block treatments and dental splints for headache);\n    * Use of opioids or barbiturates for more than 2 days\u002Fmonth within the last 4 weeks.\n11. Concurrent participation in another interventional clinical study (or within specified timeframe according to national or local legislation or requirements);\n12. Diagnosis of other significant pain disorders that could confound the assessment of headaches\u002Fmigraines or interfere with study participation, including but not limited to chronic pain disorders such as fibromyalgia, chronic low back pain and complex regional pain syndrome;\n13. Pregnant women, nursing women, premenopausal women, or WOCBP (i.e. not surgically sterile or 1 year postmenopausal) not willing to practice an acceptable contraceptive method, at the beginning of the study and for a minimum of 12 weeks following the administration of study treatment;\n14. Male subjects who are not vasectomised and who have female partners of childbearing potential and are not willing to use condoms with spermicide for a minimum of 12 weeks following the initial double-blind administration of the treatment;\n15. History of alcohol or drug abuse within 5 years of the screening visit (excluding medication overuse for headache);\n16. Body mass index (BMI) ≥35 kg\u002Fm² at the screening visit;\n17. Known clinically significant hypersensitivity to any of the study drugs, excipients or materials used to administer the study drug;\n18. Patients who, in the clinician's judgment, are actively suicidal, and therefore, deemed to be at significant risk for suicide.\n19. A diagnosis of a neuromuscular disorder or respiratory disorder, such as myasthenia gravis, Lambert-Eaton syndrome or amyotrophic lateral sclerosis that in the opinion of the investigator would compromise the safety of the study participant.",{"count":445,"type":20},641,[297],"A migraine is a headache with severe throbbing pain or a pulsating sensation, usually on one side of the head. It is often accompanied by feeling or being sick and a sensitivity to bright lights and sound. Migraines are caused by a series of events when the brain gets stimulated or activated, which causes the release of chemicals that cause pain. IPN10200 is a medication that stops the release of these chemical messengers.\n\nParticipants with episodic migraine (EM) or chronic migraine (CM) will be included in both Step 1 and Step 2. \"Headache days\" are when participants experience headaches that meet the criteria for a migraine or a headache without the additional migraine-specific symptoms. \"Migraine days\" occur when the headache displays clear migraine characteristics.\n\nThis study aims to determine:\n\n* The safety and efficacy of injecting IPN10200 directly into the muscles of the head and neck to prevent EM and CM,\n* The right amount (dose) of IPN10200 to inject at each point,\n* The total amount (dose) of IPN10200 that provides the best balance between safety and efficacy preventing migraines.\n\nParticipants will need to complete a daily electronic migraine Diary (eDiary) and questionnaires throughout the study. The total study duration for a participant will be up to 44 weeks.",[449,450],"Episodic Migraine","Chronic Migraine",{"date":429,"type":30},{"date":453,"type":30},"2024-10-10",{"date":455,"type":20},"2027-10-31",{"name":36,"class":37},166,{"id":459,"slug":460,"hasResults":11,"nctId":461,"briefTitle":462,"officialTitle":463,"acronym":464,"eligibilityCriteria":465,"healthyVolunteers":11,"sex":16,"minAge":90,"maxAge":466,"enrollmentInfo":467,"targetDuration":4,"studyType":94,"phases":469,"briefSummary":470,"conditions":471,"keywords":473,"overallStatus":75,"whyStopped":4,"lastUpdateSubmitDate":385,"lastUpdatePostDateStruct":475,"startDateStruct":476,"completionDateStruct":478,"leadSponsor":480,"locationsCount":481},"100420287","phase-1-a-study-to-assess-the-safety-and-efficacy-of-ipn10200-in-adult-participants-with-upper-limb-spasticity-100420287","NCT04752774","A Study to Assess the Safety and Efficacy of IPN10200 in Adult Participants With Upper Limb Spasticity.","An Integrated Phase I\u002FII, Multicentre, Double-blind, Randomised, Dysport and Placebo-controlled, Dose Escalation and Dose-finding Study to Evaluate the Safety and Efficacy of IPN10200 in the Treatment of Adult Upper Limb Spasticity.","LANTIMA","Inclusion Criteria:\n\n1. Participant must be 18 to 70 years of age inclusive (except for dose escalation must be 18 to 65 years of age) at the time of signing the informed consent.\n2. Has spastic hemiparesis following stroke or Traumatic brain injury (TBI)\n3. Is at least 6 months post-stroke or TBI\n4. Has never received BoNT or if previously treated, should have received their last injection of any commercialized BoNT-A or B at least 4 months prior to study Baseline\n5. Has a MAS score ≥2 in the (PTMG) to be injected\n6. Is eligible to receive a total recommended dose 1000 U Dysport in the upper limb when applicable.\n7. Has angle of spasticity ≥5° in the PTMG to be injected.\n8. Does not have any fixed contractures as defined by:\n\n   * Complete fingers extension with Angle of arrest at slow speed (Tardieu Scale) (XV1) ≥160°\n   * Complete wrist extension with XV1 ≥90°\n   * Complete elbow extension with XV1 ≥160°\n9. Physiotherapy, occupational therapy, splinting, use of benzodiazepine, and muscle relaxants had to be stable from at least 30 days preceding the study Baseline up to the Month 3 visit, and whenever possible until the end of the study.\n10. In good health (i.e. absence of any uncontrolled systemic disease or other significant medical condition) as determined by medical history, physical and neurological examinations, clinical laboratory studies, electrocardiograms (ECGs), vital signs, and Investigator's judgment prior to randomization\n11. Male and female participants Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n\nMale participants must agree that, if their partner is at risk of becoming pregnant, they will use an effective method of contraception. The participants must agree to use the contraception during the whole period of the study.\n\nA female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies: Is a woman of non-childbearing potential (WONCBP) or is a woman of childbearing potential (WOCBP) and using an acceptable contraceptive method (until the end of the study). The investigator should evaluate the potential for contraceptive method failure in relationship to the first dose of study intervention. A WOCBP must have a negative highly sensitive pregnancy test.\n\nExclusion Criteria:\n\n1. Any medical condition (including severe dysphagia or airway disease) that may increase, in the opinion of the investigator, the likelihood of adverse events (AEs) related to BoNT treatment.\n2. Known disease of the neuromuscular junction (e.g. Lambert-Eaton myasthenic syndrome, myasthenia gravis or amyotrophic lateral sclerosis etc.).\n3. Has a history of hypersensitivity to the investigational medicinal products (or other BoNTs) or any excipient used in their formulation.\n4. Clinically diagnosed significant anxiety disorder, or any other significant psychiatric disorder (e.g. depression) that might interfere with the participant's participation in the study.\n5. Likely treatment with any serotype of BoNT for any condition during the study.\n6. Undergone previous surgery to treat spasticity in the affected upper limb.\n7. Has initiated physiotherapy within 30 days prior to Baseline (if physiotherapy initiated more than 30 days prior to Baseline and ongoing, the therapy regimen should be maintained at the same frequency and intensity throughout the study if possible or at least up to 3-months post-injection).\n8. Has received previous treatment with phenol and or alcohol in the targeted upper limb any time before the study.\n9. Has been treated or is likely to be treated with intrathecal baclofen during the 30 days prior to study Baseline or during the course of the study.\n10. Current or planned treatment with any medications that interfere either directly or indirectly with neuromuscular transmission, such as curare-like non depolarising agents, lincosamides, polymyxins, anticholinesterases and aminoglycoside antibiotics, within 30 days prior to Baseline.\n11. Use of concomitant therapy which, in the investigator's opinion, would interfere with the evaluation of the safety or efficacy of the study intervention, including medications affecting bleeding disorders. For patients taking vitamin K antagonists, the INR values should be controlled (between 2 and 3)\n12. Currently planned or a history of tendon lengthening surgery, significant contracture or muscle atrophy at target joint or muscle in the past 6 months prior to Screening.\n13. Use of any experimental device within 30 days or use of any treatment with an experimental drug within five times the documented terminal half-life of the respective drug or its metabolites or if the half-life is unknown within 30 days prior to the start of the study (prior to Baseline) and during the conduct of the study.\n14. Presence of any other condition (e.g. neuromuscular disorder, muscular dystrophies, cancer cachexia, sarcopenia or other disorder that could interfere with neuromuscular function), laboratory finding or circumstance that, in the judgment of the investigator, might increase the risk to the participant or decrease the chance of obtaining satisfactory data to achieve the objectives of the study.\n15. Pregnant or lactating women, or women of childbearing potential not willing to practice a highly effective form of contraception method at the beginning of the study, for the duration of the study and for the duration of the study\n16. Inability to understand protocol procedures and requirements\n17. Infection at the injection site(s)\n18. A history of drug or alcohol abuse\n19. Male participants who are not vasectomised and who have female partners of childbearing potential and are not willing to use condoms with spermicide throughout study participation.","70 Years",{"count":468,"type":20},240,[255,297],"The purpose of the study is to assess the safety and efficacy of increasing doses of IPN10200 with the aim to evaluate the Pharmacodynamics (PD) profile of IPN10200 and to establish the total IPN10200 doses(s) that offer the best efficacy\u002Fsafety profile when used for the treatment of Adult upper limb (AUL) spasticity.",[472],"Spasticity",[474],"Upper limb spasticity after stroke or traumatic brain injury",{"date":429,"type":30},{"date":477,"type":30},"2021-04-29",{"date":479,"type":20},"2029-03-30",{"name":36,"class":37},81,""]