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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut SyndromeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Epileptic Syndromes, Lennox-Gastaut syndrome, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, hereditary disease, nervous system disorder, visual epilepsyRecruitingPhase 32026-10-09NCT05066217
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 32026-10-06NCT07416604
A Study Evaluating the Safety, Pharmacokinetics, and Activity of RO7656594 In Participants With Advanced or Metastatic Prostate CancerGenital Diseases, Male Urogenital Diseases, Neoplasms by Site, Urogenital Diseases, Urogenital Neoplasms, male reproductive organ cancer, male reproductive system disorder, metastatic prostate carcinoma, neoplasm, prostate cancer, prostate disorderRecruitingPhase 12026-10-06NCT05800665
A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of ProgressionFemale Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, IgA glomerulonephritis, Male Urogenital Diseases, Urogenital Diseases, autoimmune disease, glomerulonephritis, immune system disorder, kidney disorder, nephritis, urinary system disorderActive, not recruitingPhase 32026-10-05NCT05797610
Pyruvate Kinase Deficiency Global Longitudinal RegistryCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, anemia, congenital nonspherocytic hemolytic anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, pyruvate kinase deficiency of red cellsActive, not recruitingNot stated2026-09-28NCT03481738
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Nutritional and Metabolic Diseases, Wilson disease, basal ganglia disorder, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, inherited neurodegenerative disorder, liver disorder, metabolic disease, movement disorder, nervous system disorder, neurodegenerative diseaseActive, not recruitingPhase 1 / Phase 22026-09-25NCT04884815
A Study of Zanidatamab in Combination With Chemotherapy Plus or Minus Tislelizumab in Patients With HER2-positive Advanced or Metastatic Gastric and Esophageal CancersDigestive System Diseases, Digestive System Neoplasms, Esophageal Neoplasms, Head and Neck Neoplasms, Neoplasms by Site, autosomal dominant Parkinson disease 4, digestive system disorder, digestive system neoplasm, esophageal adenocarcinoma, esophageal disorder, gastric neoplasm, gastroesophageal adenocarcinoma, neoplasm, stomach disorderActive, not recruitingPhase 32026-09-24NCT05152147
A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia ACongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorderRecruitingPhase 32026-09-23NCT07416526
A Study With Eptinezumab in Adolescents (12-17 Years) With Chronic MigraineChronic Migraine in ChildrenRecruitingPhase 32026-08-21NCT04965675
Study to Determine if BHV-7000 is Effective and Safe in Adults With Refractory Focal Onset EpilepsyDrug Resistant Epilepsy, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, focal epilepsy, nervous system disorder, visual epilepsyActive, not recruitingPhase 2 / Phase 32026-08-13NCT06309966
Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 MutationHemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmActive, not recruitingPhase 32026-06-29NCT03173248
Study of Seladelpar in Participants With Primary Biliary Cholangitis (PBC)Digestive System Diseases, Fibrosis, Pathologic Processes, Pathological Conditions, Signs and Symptoms, bile duct disorder, biliary tract disorder, cholestasis, cirrhosis of liver, intrahepatic cholestasis, liver disorder, primary biliary cholangitisActive, not recruitingPhase 32026-05-04NCT03301506
Long-term Safety and Tolerability of BHV-7000Drug Resistant Epilepsy, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, brain disorder, central nervous system disorder, epilepsy, focal epilepsy, nervous system disorder, visual epilepsyEnrolling by invitationPhase 22026-05-01NCT06443463
TAK-788 as First-Line Treatment Versus Platinum-Based Chemotherapy for Non-Small Cell Lung Cancer (NSCLC) With EGFR Exon 20 Insertion MutationsNeoplasms by Site, Thoracic Neoplasms, bronchial neoplasm, bronchogenic carcinoma, lung disorder, lung neoplasm, neoplasm, non-small cell lung carcinoma, respiratory system disorder, respiratory tract neoplasmActive, not recruitingPhase 32026-03-30NCT04129502
A Study of TAK-861 for the Treatment of Selected Central Hypersomnia Conditionsnarcolepsy-cataplexy syndromeRecruitingPhase 2 / Phase 32026-03-05NCT05816382
Study of Nab-Paclitaxel and Gemcitabine With or Without SBP-101 in Pancreatic CancerDigestive System Diseases, Digestive System Neoplasms, Neoplasms by Site, endocrine system disorder, malignant endocrine neoplasm, neoplasm, pancreas disorder, pancreatic ductal adenocarcinoma, pancreatic neoplasmRecruitingPhase 2 / Phase 32024-11-06NCT05254171