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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
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)Hemic and Lymphatic Diseases, Neoplasms by Histologic Type, acute myeloid leukemia, hematologic disorder, leukemia, myeloid leukemia, neoplasmRecruitingPhase 32026-08-24NCT05183035
Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, bone disorder, endocrine system disorder, hereditary disease, hypochondroplasia, musculoskeletal system disorder, osteochondrodysplasiaActive, not recruitingPhase 32026-01-20NCT06455059
Extension Study of Infigratinib in Children With Achondroplasia (ACH)Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, achondroplasia, bone disorder, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasiaEnrolling by invitationPhase 22025-10-31NCT05145010
A Multicenter Multinational Observational Study of Children With HypochondroplasiahypochondroplasiaRecruitingNot stated2025-04-03NCT06212947