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
- London and Newcastle, United KingdomSee the 2 sites
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 United Kingdom
- Great Ormond Street Hospital & University College London HospitalLondon, United KingdomTaking people
- Institute of Genetic MedicineNewcastle, United KingdomTaking people
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.