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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care TreatmentBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderRecruitingPhase 32026-10-08NCT06247085
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary HemochromatosisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, hereditary disease, inborn errors of metabolism, inborn metal metabolism disorder, iron metabolism disease, iron poisoning, metabolic diseaseRecruitingPhase 22026-09-16NCT07332091
A Prospective Randomised Study of Treatment Selection Based on Epigenetic Markers Versus Standard of Care Treatment Selection in Adults With CROHN's DiseaseCrohn disease, Digestive System Diseases, digestive system disorder, gastroenteritis, inflammatory bowel disease, intestinal disorderRecruitingNot applicable2026-06-01NCT07363044
A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, brain disorder, central nervous system disorder, connective tissue disorder, hereditary disease, homocystinuria, hyperhomocysteinemia, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorderEnrolling by invitationPhase 32026-05-28NCT06431893
Personalized Medicine for Advanced Biliary Cancer PatientsBiliary Tract Neoplasms, Digestive System Diseases, Digestive System Neoplasms, Neoplasms by Site, biliary tract disorder, neoplasmRecruitingPhase 32026-05-19NCT05615818
Nivolumab in Patients With Type B3 Thymoma and Thymic Carcinoma (NIVOTHYM)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, Neoplasms by Site, Thoracic Neoplasms, Thymus Neoplasms, lymphatic system disorder, mixed neoplasm, neoplasm, thymoma, thymoma type B3Active, not recruitingPhase 22026-05-15NCT03134118