Astellas Gene Therapies | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorder	Active, not recruiting	Not applicable	2026-10-08		NCT06150820
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition	congenital structural myopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Recruiting	Not stated	2026-09-30		NCT06581146
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators	congenital structural myopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Recruiting	Phase 1 / Phase 2	2026-09-03		NCT07052929
A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)	congenital structural myopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Active, not recruiting	Phase 2 / Phase 3	2026-09-03		NCT03199469
Gene Transfer Study in Patients With Late Onset Pompe Disease	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorder	Active, not recruiting	Phase 1 / Phase 2	2026-09-02		NCT04174105
Myotubular and Centronuclear Myopathy Patient Registry	Myotubular Myopathy, X-Linked, centronuclear myopathy, congenital structural myopathy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neuromuscular disease	Recruiting	Not stated	2025-04-09		NCT04064307