Clinical trials

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. The 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.

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 \[LMP\]). Information 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. The 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. The 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. Participation is voluntary, and written informed consent will be obtained before enrollment.

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. PFIC 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). 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. 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. The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.

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. In 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. During 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. Following 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.

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. This 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.

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. This study will collect information to see how well and how safe odevixibat is in the long run for participants in South Korea.

This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives. Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching. The 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.

This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting. PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged. The 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. In 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).

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/or at any time during lactation (up to 12 months of infant age or weaning, whichever comes first. This 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. Study start date is either start of data collection or first patient enrolled whatever occurs earlier. The 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. No additional laboratory tests or HCP assessments will be required as part of this surveillance program.

The participants in this registry study will have fibrodysplasia ossificans progressiva (FOP). FOP 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)). HO is often preceded by painful, recurrent episodes of soft tissue swelling (flare-ups). This 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. The 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. This registry study will also describe the effectiveness of palovarotene in exposed participants, including the effect on everyday activities and physical performance. In 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.

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver). PBC 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. The 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. This 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. The 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). This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.

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. In this study, all participants will receive tovorafenib orally once weekly. There will be four periods in this study: 1. 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. 2. Treatment period (up to 24 months): All eligible participants will receive tovorafenib. 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. 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. 3. End-of-Treatment Safety Follow-Up (30 days): Participants will have a clinic visit 30 days after stopping treatment to check their health. 4. 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. During 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. Once tovorafenib becomes approved and commercially available in Japan, participants may transition to the commercial drug for continued treatment. A participant may withdraw consent to participate at any time.

This study will assess how well cabozantinib works and how safe it is in adults with a type of cancer called neuroendocrine tumors (NETs). These 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. The study will take place in regular hospitals and clinics in Germany and Austria. It 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. This 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.

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. Study Phases: * 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). * 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). Study Periods: Screening: Up to 28 days before first IPN60300 injection to determine eligibility. Treatment: 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. Participants will undergo blood tests, urine collections, physical examinations, and clinical evaluations.

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. Advanced 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. There will be two parts to this study: * 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. * 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. An additional part (phase II) may be added to the study based on the results of phase Ia and phase Ib. Each part will consist of the following periods: * A screening period (up to 28 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre. * 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. There 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. In 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. Each 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.

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. The 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.

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). In 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. There will be 4 periods in this study: * A screening period (up to 28 days) to assess whether the participant can take part requiring at least 1 visit to the study center. * 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. * 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. * 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. Participants 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. The 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.

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. In 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: * A screening period (up to 20 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre. * A treatment period (Day 1) where all the eligible participants will receive a single injection of IPN10200 or placebo. * 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. Participants 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.

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. All 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. There will be 3 periods in this study: * A screening period (up to 20 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre. * 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. * A follow-up period (24 weeks) after the last injection where participants' health will be monitored. Participants 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 107 weeks. Participants may withdraw consent to participate at any time.

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. CD 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. The 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. The study will consist of two periods: 1. 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. 2. A Treatment Period of 36 weeks. On 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. There 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/neurological examinations, and clinical evaluations. Participants will also need to complete questionnaires throughout the study. The total study duration for a participant will be up to 40 weeks (approximately 9 months).

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. Participants 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. This study aims to determine: * The safety and efficacy of injecting IPN10200 directly into the muscles of the head and neck to prevent EM and CM, * The right amount (dose) of IPN10200 to inject at each point, * The total amount (dose) of IPN10200 that provides the best balance between safety and efficacy preventing migraines. Participants 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.

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/safety profile when used for the treatment of Adult upper limb (AUL) spasticity.