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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving FilsuvezCollagen Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Skin Abnormalities, Skin and Connective Tissue Diseases, connective tissue disorder, epidermolysis bullosa, epidermolysis bullosa dystrophica, hereditary disease, hereditary skin disorder, junctional epidermolysis bullosa, skin disorder, vesiculobullous skin diseaseRecruitingNot stated2026-09-28NCT06423573
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingPhase 2 / Phase 32026-07-21NCT05693142
ARPKD Database StudyAbnormalities, Multiple, Agenesis of Cerebellar Vermis, Bardet-Biedl syndrome, Caroli disease, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Digestive System Abnormalities, Digestive System Diseases, Eye Diseases, Hereditary, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Male Urogenital Diseases, Meckel syndrome, type 1, Polycystic Kidney, Autosomal Recessive, Urogenital Diseases, bile duct cyst, bile duct disorder, biliary tract disorder, brain disorder, central nervous system disorder, cystic kidney disease, eye disorder, hereditary disease, hypothalamic disorder, isolated congenital hepatic fibrosis, kidney disorder, nephronophthisis, nephronophthisis 1, nervous system disorder, polycystic kidney disease, retinitis pigmentosa, urinary system disorderRecruitingNot stated2026-06-15NCT01401998
Pediatric Radio Frequency Coils GenericCongenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, Musculoskeletal Abnormalities, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingNot stated2026-06-12NCT01633866
SARC038: Phase 2 Study of Regorafenib and Nivolumab in OsteosarcomaNeoplasms by Histologic Type, Neoplasms, Bone Tissue, Neoplasms, Connective and Soft Tissue, connective tissue neoplasm, neoplasm, osteosarcoma, sarcomaActive, not recruitingPhase 22025-10-31NCT04803877