RECRUITING Phase 2 / Phase 3 ClinicalTrials.gov

A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy

Sponsor: Genethon • ClinicalTrials.gov ID: NCT07856329

Registry updated: 2026-10-13

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Description

GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin. Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leading to absence of functional dystrophin, a key protein that prevents muscle cell damage during physiological contractions. To address the underlying cause of the disease, GNT0004 delivers an optimized dystrophin gene that is capable to produce a shortened but functional dystrophin protein in target tissues (heart and skeletal muscles) in DMD subjects. It is expected to significantly delay or slow disease progression. GNT0004 will be given as a one-time intravenous (IV) infusion to help it reach muscles throughout the body.

Interventions

GNT0004 (1x10e13 vg/kg) (Genetic)
Single intravenous administration of GNT0004
GNT0004 (3x10e13 vg/kg) (Genetic)
Single intravenous administration of GNT0004
Placebo (Genetic)
Single intravenous administration of GNT0004 placebo (IMP placebo)

Eligibility criteria

Inclusion Criteria: 1. Ambulant male. 2. Being included in the GNT-014-MDYF study. 3. 6 to 10 years (inclusive). 4. For participants enrolled in Part 1: BMI scale ≤ 75th percentile. For participants enrolled in Part 2: Body weight ≤ 95th percentile of the BMI or body weight scale. 5. Positive genetic testing with detailed genotyping confirmation of DMD. 6. Able to achieve a score in the North Star Ambulatory Assessment (NSAA) scale ≥ 18 at the screening visit. 7. Stable or descending evolution in the NSAA total score during the last assessment periods recorded in study GNT-014-MDYF. 8. Gowers test (Time to Rise From Floor) ≤ 7 sec. 9. Ongoing corticosteroid therapy according to standard of care: for at least 6 months before the screening. 10. Up to date vaccination record. 11. Willing and able to comply with all protocol requirements and procedures. 12. Signed informed consents and assent as required by local law. 13. Affiliated/beneficiary of an insurance health care scheme or National Health service (if required by local law). Exclusion Criteria: 1. DMD patients with any mutations affecting: exons 1 through 17, for Part 1 participants; affecting exons 8 and/or 9 for Part 2 participants. 2. Presence of neutralizing antibodies against AAV8. 3. Cardiomyopathy based on physical/cardiological examination and echocardiography (or cardiac MRI if available). 4. Any respiratory assistance needed including non-invasive daytime or nocturnal ventilation. 5. Inability to perform the planned respiratory functions tests. 6. Any co-morbidity (ies) and/or previous or planned surgical event(s), which may interfere with DMD evolution /or evaluation of patient's safety or Investigational Medicinal Product (IMP)'s tolerability. 7. Acute infection that is not expected to be fully resolved for a sufficient time before gene therapy administration. 8. Clinically significant laboratory abnormality values as per protocol. 9. Inability to cooperate with muscle strength and functional assessments. 10. Nuclear Magnetic Resonance Imaging (MRI) contraindications: metal implants, or other contraindication for MRI such as severe phobia. 11. Unwilling and/or unable to comply with all the study protocol requirements and/or procedures. 12. Chronic hepatitis B or C, i.e., positive hepatitis B surface antigen or hepatitis C RNA viral load positive. 13. Previous inclusion to another clinical trial with any non-gene therapy related. 14. Previously treated with any drugs that may interfere with either natural diasease course and/or IMP evaluation (except for corticosteroid therapy), the last dose administration less than protocol defined washout period. 15. Received any of the treatment designed to increase dystrophin expression (e.g., exon skipping or stop codon read-through agents for DMD) within protocol defined period prior to screening. 16. Previous gene therapy for DMD.

Conditions

Duchene Muscular Dystrophy

Locations

Hôpital Universitaire des Enfants Reine Fabiola — Brussels, Belgium
UZ Leuven — Leuven, Belgium
Hôpital de la Citadelle — Liège, Belgium
University Hospital of Bordeaux — Bordeaux, France
Brest University Hospital Centre — Brest, France
Hôpital Femme Mère Enfant — Bron, France
CHU Lille — Lille, France
Centre Hospitalier Universitaire De Montpellier — Montpellier, France
Institut I-Motion, Hôpital Armand Trousseau — Paris, France
Hôpitaux Universitaires de Strasbourg — Strasbourg, France
Hospital Sant Joan de Déu Barcelona — Esplugues de Llobregat, Spain
Hospital Universitario y Politécnico La Fe — Valencia, Spain
Great Ormond Street Hospital for Children — London, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust — Newcastle upon Tyne, United Kingdom

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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