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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC DeficiencyBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Hyperammonemia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, liver disorder, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorderActive, not recruitingPhase 32026-09-30NCT06255782
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Hyperammonemia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, X-linked disease, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, liver disorder, metabolic disease, nervous system disorder, ornithine carbamoyltransferase deficiency, urea cycle disorderRecruitingNot stated2026-08-31NCT06805695
International Registry of Congenital Portosystemic Shunt (IRCPSS)46,XX disorder of sex development, Brain Diseases, Metabolic, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Digestive System Neoplasms, Disease Attributes, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hepatic Insufficiency, Hyperammonemia, Hyperandrogenism, Insulin Resistance, Liver Neoplasms, Male Urogenital Diseases, Neoplasms by Site, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, Urogenital Abnormalities, Urogenital Diseases, adrenogenital syndrome, brain disorder, cardiovascular disorder, central nervous system disorder, congenital portosystemic shunt, disorder of sexual differentiation, endocrine system disorder, glucose metabolism disease, gonadal disorder, hepatic encephalopathy, hepatopulmonary syndrome, hyperinsulinism, hypertensive disorder, hypoglycemia, liver disorder, liver failure, lung disorder, metabolic disease, neoplasm, nervous system disorder, pulmonary hypertension, respiratory system disorder, vascular disorder, vascular malformationEnrolling by invitationNot stated2025-06-18NCT06041906
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and PediatricsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Hyperammonemia, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, methylmalonic acidemia, propionic acidemiaRecruitingNot stated2025-04-15NCT05040178