Genethon
Genethon
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Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy | Duchene Muscular Dystrophy | Recruiting | Phase 2 / Phase 3 | 2026-10-02 | NCT07856329 | |
| Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin. | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, hereditary disease | Not yet recruiting | Not applicable | 2026-06-08 | NCT07633951 | |
| Natural History of Duchenne 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 | Recruiting | Not stated | 2026-02-12 | NCT03882827 | |
| Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crigler-Najjar syndrome, Nutritional and Metabolic Diseases, hereditary disease, hereditary hyperbilirubinemia, inborn errors of metabolism, metabolic disease | Recruiting | Phase 2 | 2024-12-03 | NCT06518005 | |
| Gene Therapy for Severe Crigler Najjar Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crigler-Najjar syndrome, Nutritional and Metabolic Diseases, hereditary disease, hereditary hyperbilirubinemia, inborn errors of metabolism, metabolic disease | Recruiting | Not applicable | 2023-03-28 | NCT03466463 | |
| Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, blood coagulation disease, hematologic disorder, hemorrhagic disease, hereditary disease, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopenia | Active, not recruiting | Phase 1 / Phase 2 | 2021-06-03 | NCT02333760 |