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

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

This is a longitudinal observational cohort that follows ambulatory boys with genetically confirmed Duchenne muscular dystrophy using standardized assessments such as the North Star Ambulatory Assessment, 6‑Minute Walk Test, and timed Gowers maneuver. Participants are seen regularly over a 6 to 36 month period at specialist centers in Belgium to document disease progression. The protocol aims to identify clinical, imaging, and laboratory measures that predict short‑term course and to determine the best outcome measures for future interventional trials. Data collected will serve as a baseline period for participants who may later roll over into a gene therapy phase I/II/III program.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Male
2. 4 to 9 years old inclusive
3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).

   Related to the DMD disease:
4. Diagnosis of DMD based upon documented gene testing with detailed genotyping
5. Able to achieve at inclusion and screening visits:

   1. NSAA (North Star Ambulatory Assessment) scale \> 18 or ≥ 16 if participant is between 4 and \< 5 years old at screening and:
   2. Gowers test \< or = 7 sec and/or
   3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care prior to Screening visit

   Related to the study protocol and ICH/GCP (Good Clinical Practice) requirements:
7. Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation
8. Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)

   Exclusion Criteria:

   Subject will be excluded from enrolment into the study for any of the following reasons:

   Related to the DMD disease severity:
9. Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%
10. Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
11. Any co-morbidity (ies) and or previous or planned surgical event(s) which may interfere with DMD natural evolution and or evaluation of outcomes designed to assess DMD Natural History

    Related to specific assessments:
12. Muscle testing: inability to cooperate with
13. MRI: metal implants in regions of interest for the study

    Related to the study protocol and ICH/GCP requirements:
14. Unwilling and/or unable to comply with all the study protocol requirements and/or procedures
15. Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months or IMP washout period (whichever is longer) prior to the screening visit of the study
16. Previously treated with a gene therapy drug for DMD, such as:

    * any AAV mediated gene transfer products or any gene editing products in a clinical trial or in a clinical setting,
    * if exons skipping drug was used, the last dose of exon skipping drug within 5 half-lives prior to the screening visit
17. Concomitant participation to any other interventional clinical trial

Where it's running

• Centre Hospitalier Universitaire Brugmann
  Brussels, Belgium Recruiting
• UZ Leuven
  Leuven, Belgium Recruiting
• University Hospital of Bordeaux
  Bordeaux, France Recruiting
• Brest University Hospital Centre
  Brest, France Recruiting
• Hopital Femme Mere Enfant
  Bron, France Recruiting
• CHU Lille
  Lille, France Recruiting

Study contacts

• Principal investigator: Francesco MUNTONI, Pr — Gosh London
• Study coordinator: Francesco MUNTONI, Pr
• Email: c.griffith@ucl.ac.uk
• Phone: +44 02079052602

Similar studies

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

• VM100: a nutritional supplement to see if it improves quality of life for boys with Duchenne

  This Phase 2 study tests VM100, a nutritional drink, in boys aged 8 and up with Duchenne muscular dystrophy to see if it can improve quality of life and reduce fatigue.

  Recruiting Phase 2 University of Florida · Gainesville, Florida · NCT07766980

• KER-065 for Duchenne Muscular Dystrophy: safety and potential benefit in boys and men

  This study tests whether KER-065 is safe and can help with symptoms in males with Duchenne muscular dystrophy, including both ambulatory and nonambulatory participants who are on standard care.

  Recruiting at 2 sites Phase 2 Keros Therapeutics, Inc. · Tbilisi and 3 other locations · NCT07704099

• GNR-097 gene therapy for Duchenne muscular dystrophy in boys

  This study tests a gene therapy (GNR-097) to see if it is safe, tolerable, and potentially beneficial for ambulatory boys with Duchenne muscular dystrophy.

  Recruiting at 6 sites Phase 1/2 Ao Generium · Minsk and 5 other locations · NCT07673809

• ELEVIDYS safety in Duchenne patients treated after market approval

  This study tests the safety of ELEVIDYS, with sirolimus as a protective treatment, in male children and young adults with Duchenne Muscular Dystrophy who have previously received ELEVIDYS or are eligible to receive it commercially.

  Recruiting Phase 4 Sarepta Therapeutics, Inc. · Houston, Texas · NCT07542314

• SGT-003 gene therapy for ambulant boys and young men with Duchenne muscular dystrophy (IMPACT DUCHENNE)

  This trial will test whether a single intravenous infusion of SGT-003 can help ambulatory boys and young men with Duchenne muscular dystrophy maintain or improve walking and motor function.

  Recruiting at 4 sites Phase 3 Solid Biosciences Inc. · Little Rock, Arkansas and 6 other locations · NCT07160634

• Evaluating Givinostat for Young Children with Duchenne Muscular Dystrophy

  This study is testing a new drug called Givinostat to see if it's safe and how it works in young children aged 2 to 6 with Duchenne Muscular Dystrophy.

  Recruiting at 7 sites Phase 2 Italfarmaco · Brussels and 8 other locations · NCT06769633

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.