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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 3ARecruitingPhase 2 / Phase 32026-09-22NCT02716246
Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of WoundsCollagen 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, skin disorder, vesiculobullous skin diseaseNot yet recruitingPhase 42026-08-06NCT07717736
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEBRDEBEnrolling by invitationNot stated2026-07-17NCT05708677
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing SettingCollagen 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, recessive dystrophic epidermolysis bullosa, skin disorder, vesiculobullous skin diseaseRecruitingNot stated2026-07-14NCT07700966
A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated PatientsCollagen 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, recessive dystrophic epidermolysis bullosa, skin disorder, vesiculobullous skin diseaseActive, not recruitingPhase 32025-06-27NCT05725018