Follows young boys with Duchenne muscular dystrophy to learn how the disease progresses
Follows boys aged 4 to 9 with Duchenne muscular dystrophy over 6 to 36 months.
Plain-language title generated from the registry record
Registry title: Natural History of Duchenne Muscular Dystrophy
Key facts
- Where
- Esplugues de Llobregat and Valencia, SpainSee the 2 sites
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys aged 4 to 9 years with Duchenne muscular dystrophy (DMD), confirmed by a gene test, who can still walk. It follows how the disease changes over time.
- Age
- 4 to 9 years old (inclusive)
- Sex
- Male only
- Disease stage
- DMD confirmed by documented gene testing. Must pass walking and movement tests at the visits (NSAA scale > 18, or ≥ 16 if aged 4 to under 5; Gowers test of 7 sec or less and/or 6-Minute Walk Test of 350 meters or more).
- Earlier treatment
- Must be on corticosteroid treatment, or start it as standard care before the screening visit. Must not have had gene therapy for DMD. Must not have been in a trial of an investigational medicine within the last 3 months or the washout period, whichever is longer.
- Biomarker or test result
- A DMD diagnosis based on documented gene testing with detailed genotyping.
Main reasons for not taking part
- Heart muscle disease with a heart pumping measure (LVEF) below 55%
- Needs breathing support (ventilation) by day or at night
- Other illness or surgery that could affect how DMD develops or is measured
- Cannot cooperate with muscle testing, or has metal implants in the areas scanned by MRI
- Currently in another interventional clinical trial
Read by machine on 9 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 4 and 9 years old?
Source: NCT03882827, who can take part
Where in Spain
- Hospital Sant Joan de Deu Esplugues de LlobregatEsplugues de Llobregat, SpainTaking people
- Hospital La Fe de ValenciaValencia, SpainNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Genethon
- Trial code
- NCT03882827
- Record updated
- 12 February 2026
- Original description
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.Read full original descriptionShow less
A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.
Inclusion: Male; 4 to 9 years old inclusive; Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site); Related to the DMD disease:; Diagnosis of DMD based upon documented gene testing with detailed genotyping; Able to achieve at inclusion and screening visits:; NSAA (North Star Ambulatory Assessment) scale > 18 or ≥ 16 if participant is between 4 and < 5 years old at screening and:; Gowers test < or = 7 sec and/or; 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0); 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:; Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation; Affiliated to or a beneficiary of a Health Care scheme (according to country regulation).
Exclusion: 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:; MRI: metal implants in regions of interest for the study; Related to the study protocol and ICH/GCP requirements:; 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; 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; Concomitant participation to any other interventional clinical trial; Related to the DMD disease severity:; Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%; Subject will be excluded from enrolment into the study for any of the following reasons:; Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation; Muscle testing: inability to cooperate with; Unwilling and/or unable to comply with all the study protocol requirements and/or procedures.