Association Française contre les Myopathies (AFM), Paris | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Standard of Care Comparative Arm of Phase 1/2 Gene Therapy Trial DREPAMIR" in Severe Sickle Cell Disease Patients	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease	Not yet recruiting	Not applicable	2026-08-07		NCT07752043
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function	Becker muscular dystrophy, 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 disease	Not yet recruiting	Not stated	2026-07-31		NCT07740057
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Vaso-Occlusive Crises, anemia, familial hemolytic anemia, hematologic disorder, hemolytic anemia, hereditary disease, inherited hemoglobinopathy, sickle cell disease	Recruiting	Phase 1 / Phase 2	2026-03-04		NCT07432867
Polygraphy at Home for Neuromuscular Patients Under Non-invasive Mechanical Ventilation	nervous system disorder, neuromuscular disease	Recruiting	Not stated	2025-11-24		NCT05785637
Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease	Charcot-Marie-Tooth disease, Charcot-Marie-Tooth disease type 1A, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, central nervous system malformation, hereditary disease, hereditary motor and sensory neuropathy, inherited neurodegenerative disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, polyneuropathy	Recruiting	Not applicable	2025-07-03		NCT07049588
Wheelchair Positioning and Neuromuscular Diseases	Neurologic Manifestations, Pain, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, nervous system disorder, neuromuscular disease	Recruiting	Not stated	2025-03-11		NCT05006859
Natural History Study of Children With LAMA2-related Dystrophies	Muscular dystrophy congenital, merosin negative	Recruiting	Not stated	2024-12-12		NCT06354790