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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IVBone Diseases, Metabolic, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, bone disorder, brain disorder, central nervous system disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucolipidosis, musculoskeletal system disorder, nervous system disorderEnrolling by invitationPhase 12026-02-10NCT07398872
Multicenter Randomized Controlled Trial (RCT) and Efficacy Evaluation System Study on Surgical Innovation Strategies for Neonatal Complex Congenital Heart DiseaseComplex Congenital Heart DiseaseRecruitingNot applicable2026-01-23NCT07363538
A Study in Children With AchondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasiaRecruitingNot stated2025-12-24NCT07301463
A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With AchondroplasiaBone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasiaNot yet recruitingPhase 1 / Phase 22025-12-22NCT07297875
Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic DiseasesSkin and Connective Tissue Diseases, arthritic joint disease, arthropathy, autoimmune disease, connective tissue disorder, dermatomyositis, immune system disorder, juvenile idiopathic arthritis, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease, polyarticular juvenile idiopathic arthritis, polymyositis, rheumatic disorder, skin disorder, systemic sclerosisRecruitingPhase 12025-09-19NCT07184450
The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry DiseaseBrain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorderNot yet recruitingPhase 1 / Phase 22025-02-11NCT06819514
Clinical Trial of CD19-targeted CAR-T Therapy for Refractory Juvenile DermatomyositisSkin and Connective Tissue Diseases, connective tissue disorder, dermatomyositis, muscle tissue disorder, musculoskeletal system disorder, myositis disease, nervous system disorder, neuromuscular disease, polymyositis, skin disorderRecruitingPhase 12024-08-26NCT06569472