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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
Bowel Continence Across the Lifespan in People With Spina BifidaCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Fecal Incontinence, central nervous system malformation, colonic disorder, digestive system disorder, functional colonic disease, intestinal disorder, nervous system disorder, neurogenic bowel, rectal disorder, spina bifidaRecruitingNot stated2026-09-24NCT07390318
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
LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell HistiocytosisHemic and Lymphatic Diseases, Histiocytosis, Langerhans-Cell, histiocytosis, interstitial lung disease, lung disorder, lymphatic system disorder, respiratory system disorderActive, not recruitingPhase 2 / Phase 32026-04-09NCT02205762
Improving Safety and Quality of Tracheal Intubation Practice in Pediatric ICUsFailed or Difficult Intubation, Sequela; Intubation Complication; Intubation; DifficultRecruitingNot stated2026-01-09NCT02493478
Study of Carbetocin Nasal Spray for the Treatment of Hyperphagia in Prader-Willi SyndromeHyperphagia in Prader-Willi SyndromeActive, not recruitingPhase 32025-09-10NCT06173531
National Collaborative to Improve Care of Children With Complex Congenital Heart DiseaseCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorder, hypoplastic left heart syndromeRecruitingNot stated2025-06-19NCT02852031