8 completed, 4 recruiting, 2 terminated, 2 unknown. 9 Phase 2, 7 Phase 1, 2 Not applicable, 1 Phase 3.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| 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. Dosage of AAV neutralizing antibodies - Neuromuscular Deficits, Genetic Disorder | Not applicable | Not yet recruiting | Genethon | 450 | 2026-07-31 | 2026-06-08 |
| Efficacy and Safety of GNT0003 Following Imlifidase Pre-treatment in Severe Crigler-Najjar Syndrome Imlifidase, GNT0003 - Crigler-Najjar Syndrome | Phase 2 | Recruiting | Genethon | 3 | 2024-11-08 | 2024-12-03 |
| Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome Depakine - Wolfram Syndrome | Phase 2 | Unknown | Centre d'Etude des Cellules Souches | 23 | 2021-11-26 | 2023-11-28 |
| A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy GNT0004 (1x10e13 vg/kg), GNT0004 (3x10e13 vg/kg), Placebo - Duchene Muscular Dystrophy | Phase 2 / Phase 3 | Recruiting | Genethon | 77 | 2021-03-01 | 2026-10-13 |
| Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I LGMD2I | Completed | Genethon | 52 | 2020-02-19 | 2026-02-12 | |
| Natural History of Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy | Recruiting | Genethon | 220 | 2019-12-19 | 2026-02-12 | |
| Gene Therapy for Severe Crigler Najjar Syndrome GNT0003 - Crigler-Najjar Syndrome | Not applicable | Recruiting | Genethon | 17 | 2018-03-19 | 2023-03-28 |
| Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease Lentiviral G1XCGD Gene Therapy - Granulomatous Disease, Chronic, X-linked | Phase 1 / Phase 2 | Completed | University of California, Los Angeles | 10 | 2015-10-29 | 2025-05-02 |
| Immunity Against AAV in Crigler Najjar Patient Blood sample for immunology - Crigler Najjar Syndrome | Completed | Genethon | 49 | 2014-11-01 | 2017-12-13 | |
| Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome Autologous CD34+ cells transduced with WASP lentiviral vector - Wiskott-Aldrich Syndrome | Phase 1 / Phase 2 | Active not recruiting | Genethon | 10 | 2014-09-01 | 2021-06-03 |
| Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) Myotubular Myopathy | Completed | Valerion Therapeutics, LLC | 48 | 2014-02-01 | 2018-06-07 | |
| Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) X vivo gene therapy - X-Linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Terminated | Genethon | 3 | 2013-06-24 | 2026-04-13 |
| Gene Therapy for X-linked Chronic Granulomatous Disease X vivo gene therapy - X-Linked Chronic Granulomatous Disease | Phase 1 / Phase 2 | Terminated | Genethon | 3 | 2013-06-24 | 2026-04-13 |
| Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping Duchenne Muscular Dystrophy | Unknown | Genethon | 45 | 2011-10-01 | 2016-04-12 | |
| Research of Biomarkers in Duchenne Muscular Dystrophy Patients Duchenne Muscular Dystrophy (DMD) | Completed | Genethon | 220 | 2011-06-01 | 2016-08-17 | |
| Gene Therapy for WAS Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene - Wiskott-Aldrich Syndrome | Phase 1 / Phase 2 | Completed | Genethon | 5 | 2011-05-01 | 2018-05-22 |
| Gene Therapy for Wiskott-Aldrich Syndrome (WAS) Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene - Wiskott-Aldrich Syndrome | Phase 1 / Phase 2 | Completed | Genethon | 6 | 2011-03-01 | 2021-06-03 |
| Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C AAV1-gamma-sarcoglycan vector injection - Limb Girdle Muscular Dystrophy Type 2C, Gamma-sarcoglycanopathy | Phase 1 | Completed | Genethon | 9 | 2006-11-01 | 2011-04-29 |
Crigler-Najjar Syndrome (3) · Granulomatous Disease, Chronic (3) · Muscular Dystrophy, Duchenne (3) · Wiskott-Aldrich Syndrome (3) · Neuromuscular Diseases (2) · Primary Immunodeficiency Diseases (2) · Genetic Diseases, Inborn (1) · Limb-girdle muscular dystrophy, type 2C (1) · Muscle Hypotonia (1) · Muscular Dystrophy, Limb-Girdle, Type 2I (1) · Myopathies, Structural, Congenital (1) · Rare Diseases (1) · Wolfram Syndrome (1)
Blood Specimen Collection (1) · Mac-1-like protein, Streptococcus (1) · Valproic Acid (1)
France (15) · United Kingdom (7) · Spain (4) · Belgium (3) · Italy (2) · United States (2) · Canada (1) · Denmark (1) · Germany (1) · Netherlands (1)
Adrian Thrasher, MD, PHD (1) · Caroline Y. Kuo, MD (1) · Donald B. Kohn, MD (1) · Emma Morris, MD, PHD (1) · Hal Landy, MD (1) · Hubert Serve, MD, PHD (1) · Janine Reichenbach, MD (1) · John Vissing, PR (1) · Serge Herson, Prof (1)
PMID 35075289, PMID 28716862, PMID 29664709, PMID 30902907, PMID 37585628
2009 (1), 2023 (1), P01HL073104 (1)