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Study of Microdystrophin (GNT0004) Gene Therapy for Duchenne Muscular Dystrophy in Boys Who Can Walk

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a new gene therapy for boys with Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle weakness. The treatment being tested is called GNT0004, which is a type of gene therapy designed to help the body produce a protein called microdystrophin. This protein is similar to dystrophin, which is missing or not working properly in individuals with Duchenne Muscular Dystrophy.

The study is divided into three parts. The first part aims to find the right dose of GNT0004 that is both safe and effective. Once the appropriate dose is determined, the second part of the study will compare the effects of GNT0004 with a placebo to evaluate its safety and effectiveness over a year. The final part of the study will continue to monitor the long-term safety and effectiveness of the treatment.

Participants in the study will receive the treatment through an infusion, which is a method of delivering medication directly into the bloodstream. The study will help researchers understand how well GNT0004 works in treating Duchenne Muscular Dystrophy and its potential benefits for improving muscle function in affected boys. The trial will also monitor any side effects or adverse reactions to ensure the treatment is safe for long-term use.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment will be conducted to confirm eligibility. This includes a review of medical history and a physical examination.

    Blood and urine samples will be collected to establish baseline health parameters.

  2. Step 2

    Part 1: dose determination

    In this phase, the appropriate dose of the new gene therapy, GNT0004, will be determined. This involves receiving the treatment through an intravenous infusion.

    Regular monitoring will occur to assess safety and how well the body tolerates the treatment.

  3. Step 3

    Part 2: efficacy and safety evaluation

    This phase involves comparing the effects of the determined dose of GNT0004 against a placebo. A placebo is a substance with no active treatment.

    The evaluation will last for one year, with regular visits to monitor health, including physical exams and laboratory tests.

  4. Step 4

    Medication administration

    During the trial, additional medications may be administered, such as Soliris (eculizumab) via intravenous infusion, Rapamune (sirolimus) as an oral solution, and prednisolone orally.

    The specific dosage and frequency will be determined by the study team based on individual needs and responses.

  5. Step 5

    Part 3: long-term follow-up

    After the initial evaluation period, a long-term follow-up will continue to monitor the safety and effectiveness of the treatment.

    This phase involves periodic check-ups and assessments to ensure ongoing health and well-being.

Who can join the trial?

6 criteria

  • Must be an ambulant male. (Ambulant means able to walk)
  • Must be part of the GNT-014-MDYF study.
  • Age must be between 6 to 10 years old, including both ages.
  • Body weight must be at or below the 75th percentile on the BMI scale. (BMI is a measure that uses height and weight to estimate body fat)
  • Must have a positive gene test confirming Duchenne Muscular Dystrophy (DMD). (This means a genetic test shows changes in the DMD gene that are known to cause the disease)
  • Excludes those with mutations affecting exons 1 through 17 of the DMD gene. (Exons are parts of a gene that contain the information needed to produce proteins)

Who cannot join the trial?

4 criteria

  • Patients who do not have Duchenne Muscular Dystrophy cannot participate.
  • Only male patients are eligible; female patients cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are considered part of a vulnerable population may not be eligible. A vulnerable population includes groups who may have limited ability to give informed consent or are at higher risk of harm.
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Investigated drugs

Microdystrophin (GNT0004) is a type of gene therapy being tested in this clinical trial. It is designed to help boys with Duchenne Muscular Dystrophy, a condition that weakens muscles over time. This therapy works by delivering a smaller version of the dystrophin gene, which is missing or not working properly in people with this condition. The goal is to help the body produce a protein that can strengthen and protect muscles, potentially slowing down the progression of the disease. The trial is testing different doses to find the safest and most effective amount, and it will also look at how well the therapy works and how safe it is over a longer period.

What is already known about the treatment

Microdystrophin (GNT0004) – This medication is administered as a gene therapy, specifically designed for injection into the bloodstream. It is currently being studied in clinical trials for its potential use in treating Duchenne Muscular Dystrophy, a genetic disorder characterized by progressive muscle degeneration. The main therapeutic goal is to improve muscle function and slow disease progression. At the molecular level, Microdystrophin works by delivering a modified version of the dystrophin gene to muscle cells, aiming to produce a functional protein that can help stabilize and protect muscle fibers. It is classified under gene therapies, which are a type of treatment that involves altering genes to treat or prevent disease.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. The disease typically begins in early childhood, with symptoms such as difficulty walking, frequent falls, and challenges in climbing stairs. As the condition progresses, muscle weakness spreads to the arms, neck, and other areas, leading to loss of mobility. Over time, individuals may experience complications affecting the heart and respiratory muscles. The progression of muscle weakness is gradual, impacting daily activities and overall physical function.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase I/IITrial ID2023-505187-11-00Protocol codeGNT-016-MDYFEstimated enrolment52 patientsSponsorGenethon

sourced from the EU Clinical Trials Register and site verification

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