GNT0004 gene therapy for Duchenne muscular dystrophy in ambulant boys

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The trial investigates a recombinant AAV8 gene therapy delivering a shortened but functional dystrophin gene to heart and skeletal muscles in Duchenne muscular dystrophy. It includes three parts: a dose-escalation phase to identify a safe and effective dose, a randomized blinded efficacy and safety phase comparing GNT0004 to placebo, and a long-term follow-up to monitor late effects and durability of benefit over up to five years. Participants are male, ambulant, aged 6–10, and meet genetic and functional criteria, including NSAA and Gowers test thresholds. The study uses intravenous infusion and follows standard corticosteroid therapy as background treatment."

Why it matters

Who can join: the full rules

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.

Where it's running

• Hôpital Universitaire des Enfants Reine Fabiola
  Brussels, Belgium Recruiting
• UZ Leuven
  Leuven, Belgium Recruiting
• University Hospital of Bordeaux
  Bordeaux, France Recruiting
• Brest University Hospital Centre
  Brest, France Recruiting
• Hôpital Femme Mère Enfant
  Bron, France Recruiting
• CHU Lille
  Lille, France Recruiting

Study contacts

• Study coordinator: Medical Lead
• Email: contact@genethon.fr
• Phone: +33 01 69 47 25 38

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• AOC 1044 in Duchenne Muscular Dystrophy with Exon 44 Skipping

  This study tests whether intravenous AOC 1044 helps boys with Duchenne muscular dystrophy caused by mutations amenable to exon 44 skipping.

  Recruiting at 20 sites Phase 3 Avidity Biosciences, Inc. · Westmead, New South Wales and 19 other locations · NCT07587242

• Ultrafast ultrasound biomarkers to track Duchenne and Becker muscular dystrophy

  This study tests whether multiparametric ultrafast ultrasound can non-invasively monitor disease progression in people with Duchenne or Becker muscular dystrophy.

  Recruiting Nantes University Hospital · Nantes · NCT07378553

• Real-world experience of oral givinostat for Duchenne muscular dystrophy in the U.S.

  The study will track safety and functional outcomes in people aged 6 and older with Duchenne muscular dystrophy who start oral givinostat during routine U.S. care to see how the medicine is tolerated and how patients do over time.

  Recruiting at 9 sites ITF Therapeutics LLC · Little Rock, Arkansas and 8 other locations · NCT07127978

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.