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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination TherapyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingPhase 32026-10-09NCT05444257
A Study Observing the Long-term, Effectiveness and Safety of Odevixibat (Bylvay) in Patients With Alagille Syndrome (ALGS) Who Are Receiving Ongoing TreatmentAbnormalities, Multiple, Alagille syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, bile duct disorder, biliary tract disorder, cardiovascular disorder, cholestasis, congenital heart disease, hereditary disease, intrahepatic cholestasis, liver disorderRecruitingNot stated2026-10-01NCT06850038
A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without InhibitorsCongenital, 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 disorderActive, not recruitingPhase 32026-09-18NCT05135559
Cord Clamping Among Neonates With Congenital Heart DiseaseCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiovascular disorder, congenital heart disease, heart disorderRecruitingNot applicable2026-08-12NCT06153459
TReatment for ImmUne Mediated PathopHysiologyBrain Diseases, Metabolic, Digestive System Diseases, Hepatic Insufficiency, Nutritional and Metabolic Diseases, acute liver failure, brain disorder, central nervous system disorder, hepatic encephalopathy, liver disorder, liver failure, metabolic disease, nervous system disorderActive, not recruitingPhase 22026-08-12NCT04862221
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to GrowCardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Musculoskeletal Abnormalities, Noonan syndrome, Sex Chromosome Disorders, Skin and Connective Tissue Diseases, Turner syndrome, Urogenital Abnormalities, Urogenital Diseases, cardiovascular disorder, chromosomal disorder, congenital heart disease, connective tissue disorder, disorder of sexual differentiation, endocrine system disorder, gonadal disorder, gonadal dysgenesis, heart disorder, hereditary disease, musculoskeletal system disorder, sex chromosome disorder of sex developmentActive, not recruitingPhase 32026-06-12NCT05330325
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of AgeCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderActive, not recruitingPhase 32026-05-28NCT05422222
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and OlderCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Diseases, Infant, Newborn, Diseases, cystic fibrosis, hereditary disease, lung disorder, pancreas disorder, respiratory system disorderEnrolling by invitationPhase 32026-04-29NCT05844449
FSL2 Post Approval Study for Pediatric and Young Adults PatientsNutritional and Metabolic Diseases, diabetes mellitus, endocrine system disorder, glucose metabolism disease, metabolic diseaseActive, not recruitingNot stated2026-04-09NCT04959552
Ketotifen for Children With Functional Dyspepsia in Association With Duodenal EosinophiliaEosinophilia, Hemic and Lymphatic Diseases, hematologic disorder, leukocyte disorderRecruitingPhase 32026-01-09NCT02484248