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Clinical trials and research at Genethon

• 4Clinical trials
• 6Countries

Newest trials

All recruiting trials (4)

• GNT0004 gene therapy for Duchenne muscular dystrophy in ambulant boys

  This study tests a one-time IV gene therapy (GNT0004) to see if it slows DMD progression in ambulant boys aged 6–10.

  Recruiting at 10 sites Phase 2/3 Genethon · Brussels and 13 other locations · NCT07856329

• Treatment for severe Crigler-Najjar syndrome using gene therapy

  This study is testing a new gene therapy to see if it can safely lower bilirubin levels in people with severe Crigler-Najjar syndrome and help them avoid needing a liver transplant.

  Recruiting Phase 2 Genethon · Clamart · NCT06518005

• Natural history of Duchenne muscular dystrophy in boys aged 4–9

  This project will follow boys aged 4–9 with Duchenne muscular dystrophy for 6–36 months to collect standard clinical, imaging, and lab data and create a baseline before possible gene therapy.

  Recruiting at 12 sites Genethon · Brussels and 14 other locations · NCT03882827

• Gene therapy for severe Crigler-Najjar syndrome

  This study is testing a new gene therapy called GNT0003 to see if it can safely help people aged 10 and older with severe Crigler-Najjar syndrome who need light treatment.

  Recruiting at 4 sites Genethon · Clamart and 3 other locations · NCT03466463

Quick answers

How many clinical trials run by Genethon are recruiting right now?

4, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 6 countries. The most sites are in France (4 trials), Belgium (2 trials), Spain (2 trials), United Kingdom (2 trials), Italy (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2/3: 1, Phase 2: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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