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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated CardiomyopathyCardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, laminopathy, restrictive cardiomyopathy, subvalvular aortic stenosisActive, not recruitingNot stated2026-09-02NCT05112237
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