[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Institut de Recherches Internationales Servier\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":197},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,92,127,152,174],{"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":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":59,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":84,"completionDateStruct":86,"leadSponsor":88,"locationsCount":91},"100499720","phase-1-phase-12-trial-of-s241656-in-selected-rasmapk-mutation--positive-malignancies-100499720",false,"NCT05786924","Phase 1\u002F2 Trial of S241656 in Selected RAS\u002FMAPK Mutation- Positive Malignancies","A Phase 1\u002F2, Open-label Study of Oral S241656 (BDTX-4933) as Monotherapy and in Combination With Other Anti-Cancer Therapies in Patients With KRAS, BRAF and Other Selected RAS\u002FMAPK Mutation-Positive Malignancies","Key Inclusion Criteria:\n\n* Life expectancy of ≥ 12 weeks in the opinion of the investigator.\n* Histologically or cytologically confirmed recurrent locally advanced (unresectable) or metastatic solid tumors with documented RAS or RAF mutations or alterations.\n* Adequate bone marrow and organ function.\n* Recovered from toxicity to prior anti-cancer therapy.\n\nPart 1 Dose Escalation cohort ONLY:\n\n* Part 1A: Advanced\u002Fmetastatic NSCLC with KRAS non-G12C, HRAS, NRAS, BRAF or CRAF (RAF1) mutations or alterations\n* Part 1B: Advanced\u002Fmetastatic GI tumors (e.g., PDAC, CRC, and BTC) with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations or alterations\n* Part 1C: Advanced\u002Fmetastatic PDAC with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations or alterations\n* Part 1D: Colorectal adenocarcinoma with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations or alterations\n* Part 1E: Other advanced\u002Fmetastatic non-GI, non-NSCLC solid tumors with KRAS, HRAS, NRAS, BRAF, CRAF (RAF1) mutations or alterations\n\nPart 2 Dose Optimization and Expansion cohorts ONLY:\n\n* Part 2A: Advanced\u002Fmetastatic NSCLC with KRAS non-G12C mutations and\u002For BRAF mutations\n* Part 2A1: Advanced\u002Fmetastatic NSCLC with KRAS non-G12C mutations\n* Part 2A2: Advanced\u002Fmetastatic NSCLC with BRAF mutations\n* Part 2A3: Advanced\u002Fmetastatic NSCLC with KRAS non-G12C or BRAF mutations or alterations and active CNS metastatic disease\n* Part 2A4: Advanced\u002Fmetastatic NSCLC with a KRAS G12C mutation\n* Part 2B1: Advanced\u002Fmetastatic PDAC with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations or alterations\n* Part 2B2: Advanced\u002Fmetastatic CRC with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations or alterations\n* Part 2B3: Advanced\u002Fmetastatic BTC (adenocarcinoma) with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations or alterations\n\nKey Exclusion Criteria:\n\n* Cancer that has a known MEK1\u002F2 mutation.\n* Known allergy\u002Fhypersensitivity to excipients of S241656 or to any of the registered IMPs administered in combination.\n* Any contra-indication, to use of any of the combination chemotherapy or anti-EGFR therapy partners administered as part of this trial.\n* Major surgery within 4 weeks of study entry or planned during study.\n* Ongoing anticancer therapy.\n* Ongoing radiation therapy.\n* Uncontrolled or active clinically relevant bacterial, fungal, or specific viral infection requiring systemic therapy.\n* Clinically significant cardiovascular disease.\n* Symptomatic spinal cord compression.\n* Evidence of active malignancy (other than study-specific malignancies) requiring systemic therapy within the next 2 years.\n* History or current evidence of retinal vein occlusion (RVO) or current risk factors for RVO.\n* Females who are pregnant or breastfeeding.\n* Actively receiving systemic treatment or direct medical intervention on another therapeutic clinical study.\n* Prior use of experimental agents that target the KRAS\u002FBRAF\u002FMEK\u002FERK pathway.","ALL","18 Years",{"count":19,"type":20},554,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","BDTX-4933-101 is a first-in-human, open-label, Phase 1\u002F2 dose escalation, dose optimization and expansion study designed to evaluate the safety and tolerability of S241656 as monotherapy and in combination with other anti-cancer therapies in participants with selected advanced malignancies. The study population for the Dose Escalation part of the study comprises adults with recurrent advanced\u002Fmetastatic non-small cell lung cancer (NSCLC), Gastrointestinal (GI) cancers, and other solid tumors harboring KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (Rapidly Accelerated Fibrosarcoma (RAF1)) mutations or alterations. A dose optimization part in adults with NSCLC may follow the dose escalation phase if the sponsor, in consultation with the safety review committee, decides it is necessary to further characterize the optimal dose. However, the study may also proceed directly to the expansion phase. The study population for the Dose Expansion part of the study comprises adults with advanced\u002Fmetastatic NSCLC with KRAS and\u002For BRAF mutations, and with Pancreatic Ductal AdenoCarcinoma (PDAC), ColoRectal Cancer (CRC), and Biliary Tract Cancer (BTC) with KRAS, HRAS, NRAS, BRAF, and\u002For CRAF (RAF1) mutations and alterations. All patients will self-administer S241656 orally in 28-day cycles until disease progression, toxicity, withdrawal of consent, or termination of the study.",[27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,48,49,50,51,52,53,54,55,56,57,58],"Non-small Cell Lung Cancer","Histiocytic Neoplasm","Histiocytosis","BRAF Gene Mutation","BRAF V600E","BRAF V600 Mutation","BRAF Mutation-Related Tumors","BRAF","Metastatic Lung Non-Small Cell Carcinoma","Metastatic Lung Cancer","Recurrent Lung Cancer","Recurrent Lung Non-Small Cell Carcinoma","NSCLC","Solid Tumor","Solid Carcinoma","KRAS G12D","KRAS G12V","KRAS Mutation-Related Tumors","NRAS Gene Mutation","Thyroid Cancer","Thyroid Carcinoma","Colorectal Cancer","Colorectal Carcinoma","Recurrent Histiocytic and Dendritic Cell Neoplasm","Brain Metastases","Recurrent NSCLC","KRAS G13C","Acquired Resistance to KRAS G12C Inhibitor","KRAS G12A","KRAS G12F","KRAS G12R","KRAS G13D",[60,61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78],"BRAF Class I","BRAF Class II","BRAF Class III","KRAS","Intolerant histiocytic neoplasm","BDTX-4933","Phase 1","dose escalation","dose expansion","MAPK","mitogen-activated protein kinase","RAS","RAF","Upstream oncogenic alterations","RAF inhibitor","intracranial disease","CRAF","NRAS","RAF fusions","RECRUITING","2026-06-16",{"date":82,"type":83},"2026-06-17","ACTUAL",{"date":85,"type":83},"2023-04-18",{"date":87,"type":20},"2028-06",{"name":89,"class":90},"Institut de Recherches Internationales Servier","OTHER",27,{"id":93,"slug":94,"hasResults":11,"nctId":95,"briefTitle":96,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":99,"targetDuration":4,"studyType":21,"phases":101,"briefSummary":102,"conditions":103,"keywords":108,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":118,"lastUpdatePostDateStruct":119,"startDateStruct":121,"completionDateStruct":123,"leadSponsor":125,"locationsCount":126},"100214912","phase-1-study-of-orally-administered-ag-120-in-subjects-with-advanced-hematologic-malignancies-with-an-idh1-mutation-100214912","NCT02074839","Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation","A Phase I, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects With Advanced Hematologic Malignancies With an IDH1 Mutation","Key Inclusion Criteria:\n\n* Subject must be ≥18 years of age.\n* Subjects must have documented IDH1 R132 gene-mutated advanced hematologic malignancy based on local or central evaluation.\n* Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study.\n* Subjects must have ECOG PS of 0 to 2.\n* Platelet count ≥20,000\u002FµL (Transfusions to achieve this level are allowed).\n* Subjects must have adequate hepatic function as evidenced by: Aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤3.0 × ULN, unless considered due to leukemic disease and serum total bilirubin ≤1.5 x upper limit of normal (ULN), unless considered due to Gilbert's disease or leukemic disease\n* Subjects must have adequate renal function as evidenced by a serum creatinine ≤2.0 × ULN or creatinine clearance \\>40mL\u002Fmin based on Cockroft-Gault glomerular filtration rate (GFR)\n* Subjects must be recovered from any clinically relevant toxic effects of any prior surgery, radiotherapy, or other therapy intended for the treatment of cancer.\n* Female subjects with reproductive potential must have a negative serum pregnancy test within 7 days prior to the start of therapy and on the first day of study drug administration.\n\nKey Exclusion Criteria:\n\n* Subjects who have undergone hematopoietic stem cell transplant (HSCT) within 60 days of the first dose of AG-120, or subjects on immunosuppressive therapy post HSCT at the time of screening, or with clinically significant graft-versus-host disease (GVHD). (The use of a stable dose of oral steroids post HSCT and\u002For topical for ongoing skin GVHD is permitted.)\n* Subjects who received systemic anticancer therapy or radiotherapy \\\u003C14 days prior to their first day of study drug administration. (Hydroxyurea is allowed prior to enrollment and after the start of AG-120).\n* Subjects who received an investigational agent \\\u003C14 days prior to their first day of study drug administration.\n* Subjects who are pregnant or breastfeeding.\n* Subjects with an active severe infection or with an unexplained fever \\>38.5°C during screening visits or on their first day of study drug administration (at the discretion of the Investigator, subjects with tumor fever may be enrolled).\n* Subjects with New York Heart Association (NYHA) Class III or IV congestive heart failure or LVEF \\\u003C40% by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan within approximately 28 days of C1D1.\n* Subjects with a history of myocardial infarction within the last 6 months of screening.\n* Subjects with a known unstable or uncontrolled angina pectoris.\n* Subjects with a known history of severe and\u002For uncontrolled ventricular arrhythmias.\n* Subjects with known unstable or uncontrolled angina pectoris.\n* Subjects with heart-rate corrected QT (QTc) interval ≥450 ms or other factors that increase the risk of QT prolongation or arrhythmic events.\n* Patients taking medications that are known to prolong the QT interval\n* Subjects with known infection with human immunodeficiency virus (HIV) or active hepatitis B or C.\n* Subjects with clinical symptoms suggesting active central nervous system (CNS) leukemia or known CNS leukemia. Evaluation of cerebrospinal fluid is only required if there is a clinical suspicion of CNS involvement by leukemia during screening.\n* Subjects with immediately life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, and\u002For disseminated intravascular coagulation.",{"count":100,"type":20},291,[23],"The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation. The first portion of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of AG-120 to determine maximum tolerated dose (MTD) and\u002For the recommended Phase II dose. The second portion of the study is a dose expansion phase where four cohorts of patients will receive AG-120 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose. Additionally, the study includes a substudy evaluating the safety and tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of AG-120 in subjects with relapsed or refractory myelodysplastic syndrome with an IDH1 mutation. Anticipated time on study treatment is until disease progression or unacceptable toxicity occurs.",[104,105,106,107],"Relapsed or Refractory Acute Myeloid Leukemia (AML)","Untreated AML","Other IDH1-mutated Positive Hematologic Malignancies","Myelodysplastic Syndromes",[109,110,111,112,113,114,105,115,116,117],"acute myeloid leukemia","AML","myelodysplastic syndrome","MDS","hematologic malignancies","IDH","IDH1","relapsed AML","refractory AML","2026-06-11",{"date":120,"type":83},"2026-06-15",{"date":122,"type":83},"2014-03-01",{"date":124,"type":20},"2026-12-31",{"name":89,"class":90},30,{"id":128,"slug":129,"hasResults":11,"nctId":130,"briefTitle":131,"officialTitle":132,"acronym":133,"eligibilityCriteria":134,"healthyVolunteers":11,"sex":16,"minAge":135,"maxAge":136,"enrollmentInfo":137,"targetDuration":4,"studyType":21,"phases":139,"briefSummary":140,"conditions":141,"keywords":4,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":143,"lastUpdatePostDateStruct":144,"startDateStruct":146,"completionDateStruct":148,"leadSponsor":150,"locationsCount":151},"100610459","phase-1-a-first-in-human-study-of-s230815-in-pediatric-participants-with-kcnt1-related-developmental-and-epileptic-encephalopathy-100610459","NCT07227857","A First-in-human Study of S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy","A Phase Ib\u002FII First-in-human, Multicentre, Open-label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effect of Intrathecal S230815 in Pediatric Participants With KCNT1-related Developmental and Epileptic Encephalopathy","KANDLE","Inclusion Criteria:\n\n* Male or female pediatric participants aged 2-12 years old at screening, with a genetically confirmed diagnosis of Developmental Epileptic Encephalopathy (DEE) due to a pathogenic or likely pathogenic variant in KCNT1 confirmed by central genetic testing.\n* Stable dose of other regular medications and\u002For stable antiseizure interventions (such as ketogenic diet and vagal nerve stimulation).\n\nExclusion Criteria:\n\n* Other clinical phenotypes associated with pathogenic or likely pathogenic variants in KCNT1 other than Epilepsy of Infancy with Migrating Focal Seizures or Early-Onset Epileptic Encephalopathy\n* Documented pathogenic or likely pathogenic variants in any other gene known to cause epilepsy identified through prior genetic testing. Variants of uncertain significance in other genes known to cause epilepsy may be considered on discussion with the sponsor.\n* Clinically significant medical history or clinical findings on physical examination, other than DEE, that in the judgment of the investigator, make the participant unsuitable for participation in the study and\u002For completion of the trial procedures, including, but not limited to:\n\n  * Clinically significant prior or ongoing medical conditions within 30 days of the screening visit, as per investigator judgement.\n  * Clinically significant abnormality on Electrocardiogram (ECG) at the screening visit, as per investigator judgement.\n  * Clinically significant abnormality on laboratory testing at screening, including, but not limited to:\n  * Renal insufficiency, which is defined as creatinine clearance \\\u003C 40 mL\u002Fmin assessed as estimated glomerular filtration rate (eGFR) using Modification of Diet in Renal Disease (MDRD) formula\n  * Hepatic derangement defined as transaminase values more than 3 times the Upper Limit of Normal (ULN) range, or total bilirubin values more than 1.5 times the ULN.\n* Positive hepatitis B surface antigen test, positive hepatitis C antibody test, positive for human immunodeficiency virus (HIV), as reported by a laboratory test within 6 months prior to the screening visit, or on screening bloods.\n* Bone, spine, bleeding disorders, or other disorder that exposes the participant to risk of injury or unsuccessful Lumbar puncture (e.g., haemophilia, Von Willebrand's disease, liver disease).\n* Contraindications to undergoing Magnetic Resonance Imaging (MRI), Lumbar puncture procedure and Intrathecal administration.\n* History of Central Nervous System (CNS) tumors or malignancies, including CNS metastatic disease.\n* Continuous respiratory support, defined as oxygen supplementation or non-invasive ventilation (e.g.: continuous positive airway pressure, bi-level intermittent positive airway pressure), required during waking hours. This does not include suctioning; cough assist devices or other devices that may be used regularly to clear airways.\n* Invasive ventilation including the presence of a tracheostomy.\n* Use of quinidine within 30 days prior to the screening visit.\n* Current use or anticipated use of antiplatelet or anticoagulant therapy during the study.\n* Current or past enrolment in an interventional clinical study in which an investigational therapy is\u002Fwas administered within 30 days (or 5 half-lives of study agent, whichever is longer) prior to the screening visit.\n* Implantable CNS device that may interfere with the ability to administer the study drug via Lumbar puncture.\n* Known hypersensitivity to any oligonucleotide, as demonstrated by a systemic allergic reaction (e.g., changes in pulse, blood pressure, breathing function, etc.), or any other drug that in the opinion of the investigator may preclude study participation.","2 Years","12 Years",{"count":138,"type":20},20,[23,24],"Study CL1-230815-001 (KANDLE) is a Phase Ib\u002FII, First In Human, multicentre, open-label, multiple ascending dose study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamic (PD) effect of S230815 in pediatric participants with KCNT1-related Developmental Epileptic Encephalopathy. To participate in the study, participants must have a diagnosis of Developmental Epileptic Encephalopathy due to a documented pathogenic or likely pathogenic variant in KCNT1 (to be confirmed by central genetic testing at the screening visit). The study consists of a screening period followed by two consecutive interventional parts. Part 1 will evaluate multiple ascending doses of S230815. Part 2 is a long-term treatment extension for participants who have completed Part 1. Participants will seamlessly roll-over from Part 1 to Part 2, resuming the same cohort as they were assigned in Part 1, and will receive S230815 for a maximum of 72 weeks.",[142],"Epileptic Encephalopathy","2026-05-21",{"date":145,"type":83},"2026-05-26",{"date":147,"type":83},"2025-11-24",{"date":149,"type":20},"2028-04-15",{"name":89,"class":90},16,{"id":153,"slug":154,"hasResults":11,"nctId":155,"briefTitle":156,"officialTitle":157,"acronym":4,"eligibilityCriteria":158,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":159,"targetDuration":4,"studyType":21,"phases":161,"briefSummary":162,"conditions":163,"keywords":4,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":168,"completionDateStruct":170,"leadSponsor":172,"locationsCount":173},"100554633","phase-1-ivosidenib-plus-durvalumab-and-gemcitabinecisplatin-as-first-line-therapy-in-participants-with-locally-advanced-or-metastatic-cholangiocarcinoma-with-an-idh1-mutation-100554633","NCT06501625","Ivosidenib Plus Durvalumab and Gemcitabine\u002FCisplatin as First-Line Therapy in Participants With Locally Advanced or Metastatic Cholangiocarcinoma With an IDH1 Mutation","A Phase 1b\u002F2, Safety Lead-in and Dose-Expansion, Open Label, Multicenter Trial Investigating the Safety, Tolerability, and Preliminary Activity of Ivosidenib in Combination With Durvalumab and Gemcitabine\u002FCisplatin as First-line Therapy in Participants With Locally Advanced, Unresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation","Inclusion Criteria:\n\n* Have a histopathological confirmed diagnosis consistent with locally advanced unresectable or metastatic cholangiocarcinoma.\n* Have documented IDH1 gene-mutated cholangiocarcinoma based on local or central laboratory testing (R132C\u002FL\u002FG\u002FH\u002FS mutation variants tested).\n* Have at least one evaluable and measurable lesion as defined by RECIST v1.1.\n* Have adequate bone marrow function as evidenced by:\n* Absolute neutrophil count ≥ 1,500\u002Fmm3 or 1.5 ×109\u002FL\n* Hemoglobin ≥ 9 g\u002FdL\n* Platelet count ≥ 100,000\u002Fmm3 or 100 × 109\u002FL\n* Have adequate hepatic function as evidenced by:\n* Serum bilirubin ≤ 2.0 × the upper limit of normal (ULN); this will not apply to patients with confirmed Gilbert's syndrome. Any clinically significant biliary obstruction should be resolved before randomization\n* Aspartate aminotransferase (AST), and alanine aminotransferase (ALT) ≤ 2.5 × ULN; for patients with hepatic metastases, ALT and AST ≤ 5.0 × ULN\n* Have adequate renal function, defined as: creatinine clearance \\> 60 mL\u002Fmin per 24 hour urine or as calculated on the Cockcroft-Gault formula (using actual body weight):\n\nCreatine CL (mL\u002Fmin)= (140 - Age) × (weight in kg) × (0.85 if female)\u002F72 × serum creatinine (mg\u002FdL)\n\nExclusion Criteria:\n\n* Received treatment for locally advanced, unresectable or metastatic disease with the following exceptions:\n* Treatment with up to one cycle of durvalumab plus gemcitabine\u002Fcisplatin treatment is permitted before study participation. Note: For the Safety Lead-In Phase, participants who received one prior cycle of durvalumab plus gemcitabine\u002Fcisplatin and required dose modifications for treatment-related toxicity are excluded.\n* Patients who developed recurrent disease \\> 6 months after surgery with curative intent, and, if given, \\> 6 months after the completion of adjuvant (chemotherapy and\u002For radiation).\n* Prior exposure to immune-mediated therapy, including, but not limited to, anti-PD-1or other anti-PD-L1, and anti-PD-L2, anti-CTLA-4 antibodies, excluding therapeutic anticancer vaccines.\n* Unresolved Grade ≥2 adverse events from a previous anticancer therapy, with the exception of alopecia and vitiligo and the laboratory values listed in the inclusion criteria.\n* Patients with Grade ≥2 neuropathy to be evaluated on a case-by-case basis after consultation with the medical monitor\n* Patients with irreversible toxicity not reasonably expected to be exacerbated by treatment with ivosidenib may be included only after consultation with the medical monitor\n* Participation in another interventional study at the same time or within 14 days prior to the first study medication (triple combination treatment) administration. For patients having participated to another prior interventional study, the first dose of ivosidenib should occur after a period greater than or equal to 5 half-lives or 28 days, whichever is shorter of the last dose of the prior investigational product.\n* Active or prior documented autoimmune or inflammatory disorders including:\n* inflammatory bowel disease (e.g., colitis or Crohn's disease)\n* diverticulitis (with the exception of diverticulosis)\n* systemic lupus erythematosus\n* Sarcoidosis syndrome\n* Wegener syndrome (granulomatosis with polyangiitis, Graves' disease, rheumatoid arthritis, hypophysitis, uveitis, etc.)\n\nNote: in cases with no active disease for ≥ 5 years, patients may be considered for inclusion if approved by the Medical Monitor. Participants with the following conditions are eligible for the study:\n\n* chronic skin condition that does not require systemic therapy\n* vitiligo\n* alopecia\n* hypothyroidism (e.g., following Hashimoto syndrome) stable on hormone replacement therapy\n* unmedicated celiac disease that is controlled by diet\n* Have heart rate-corrected QT interval using Fridericia's formula (QTcF) of ≥ 450 msec or with other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome\u002Fsudden death, polymorphic ventricular arrhythmia). The Sponsor should review participants with bundle branch block and prolonged QTcF for potential inclusion.\n* Have an active infection, including:\n* Hepatitis B (clinical evaluation includes: presence of hepatitis B surface antigen \\[HBsAg\\] and\u002For anti-HBcAb with detectable hepatitis B virus \\[HBV\\] DNA ≥ 10 IU\u002FmL)\n* Hepatitis C\n* Tuberculosis (clinical evaluation includes: clinical history, physical examination and\u002For radiographic findings, and tuberculosis testing as per local practice)\n* Human immunodeficiency virus (clinical evaluation includes: positive HIV 1\u002F2 antibodies) Note: Patients with a resolved or past HBV infection (i.e., presence of hepatitis B core antibody \\[anti-HBc\\] and absence of HBsAg) do not need to be excluded from the study. Patients positive for hepatitis C (HCV) antibody are eligible only if the polymerase chain reaction is negative for HCV RNA.",{"count":160,"type":20},52,[23,24],"The objective of this study is to investigate the safety, tolerability and preliminary activity of ivosidenib in combination with durvalumab and gemcitabine\u002Fcisplatin as first-line therapy in participants with locally advanced, unresectable or metastatic cholangiocarcinoma with an IDH1 mutation. The study will begin with a safety lead-in phase (Phase 1b study) to determine the recommended combination dose (RDC) and then will transition to an expansion phase (Phase 2 study) to assess the clinical activity of ivosidenib in combination with durvalumab and gemcitabine\u002Fcisplatin at the RCD. During the treatment period participants will have study visits on days 1, 8, and 15 of Cycle 1, on days 1 and 8 of Cycle 2 to 8, and on day 1 of each additional cycle. Cycles 1 through 8 are 21 day cycles, and each following cycle is 28 days. Approximately 30 days and 90 days after treatment has ended, safety follow-up visits will occur and then participants will be followed for survival every 3 months. Study visits may include blood tests, ECG, vital signs, and a physical examination.",[164],"Locally Advanced, Unresectable or Metastatic Cholangiocarcinoma With an IDH1 Mutation","2026-05-08",{"date":167,"type":83},"2026-05-11",{"date":169,"type":83},"2024-12-16",{"date":171,"type":20},"2027-09-13",{"name":89,"class":90},38,{"id":175,"slug":176,"hasResults":11,"nctId":177,"briefTitle":178,"officialTitle":179,"acronym":180,"eligibilityCriteria":181,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":182,"targetDuration":4,"studyType":21,"phases":184,"briefSummary":186,"conditions":187,"keywords":4,"overallStatus":79,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":190,"startDateStruct":191,"completionDateStruct":193,"leadSponsor":195,"locationsCount":196},"100551889","phase-3-ivosidenib-ivo-monotherapy-and-azacitidine-aza-monotherapy-in-patients-with-hypomethylating-agent-hma-naive-myelodysplastic-syndromes-mds-with-an-idh1-mutation-100551889","NCT06465953","Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an IDH1 Mutation","A Phase 3, Multicenter, Open Label, Randomized, Non-comparative Two-arm Study of Ivosidenib (IVO) Monotherapy and Azacitidine (AZA) Monotherapy in Adult Patients With Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS) With an Isocitrate Dehydrogenase-1 (IDH1) Mutation (PyramIDH Study)","PyramIDH","Inclusion Criteria:\n\n* Diagnosis of HMA naive IDH1 R132 mutated MDS defined according to WHO criteria (5th edition):\n* Moderate high, high and very high-risk MDS per IPSS-M score will be eligible regardless of blood counts and with blast counts 0-19%.\n* Low and moderate low-risk MDS per IPSS-M score must:\n* Have cytopenias related to MDS, defined as: \\\u003C100 platelets\u002Fmicroliter, or absolute neutrophil count (ANC) \\\u003C1000\u002Fmm3, or hemoglobin \\\u003C10g\u002FdL AND\n* Have a blast count between 5-19% AND\n* Be eligible for HMA therapy (very low risk participants are to be excluded)\n* Locally or centrally confirmed IDH1 R132 C\u002FG\u002FH\u002FL\u002FS mutation\n\nExclusion Criteria:\n\n* Received prior anticancer\u002Fdisease modifying treatment for MDS (including HMA's, cytotoxic chemotherapy, investigational agents, bcl-2 inhibitor based-regimens, hematopoietic stem cell transplant (HSCT), IDH1 inhibitors). For LR-MDS patients, prior treatment with growth factors, luspatercept, lenalidomide, and imetelstat are allowed.\n* \\>20% blasts by morphology or immunohistochemistry on screening bone marrow aspirate\u002Fbiopsy",{"count":183,"type":20},48,[185],"PHASE3","This study will enroll participants with myelodysplastic syndromes (MDS) with an Isocitrate dehydrogenase protein, 1 (IDH1) mutation, who have not received treatment with a hypomethylating agent previously. Participants will be randomized to receive either ivosidenib (IVO) alone or azacitidine (AZA) alone. IVO will be administered daily throughout the 28-day treatment cycle and AZA will be administered for the first 7 days of each 28-day cycle. Study visits will be conducted every week during Cycle 1 (Days 1, 8, 15, and 22), and Day 1 of each cycle thereafter. After the last dose of treatment, participants will attend an safety follow-up visit and participants will be followed to assess overall survival. Study visits may include a bone marrow aspirate, physical exam, echocardiogram (ECHO), electrocardiogram (ECG), blood and urine analysis, and questionnaires.",[188,189],"Hypomethylating Agent (HMA) Naive Myelodysplastic Syndromes (MDS)","Myelodysplastic Syndromes (MDS)",{"date":167,"type":83},{"date":192,"type":83},"2024-12-03",{"date":194,"type":20},"2028-12-01",{"name":89,"class":90},62,""]