An observational study in Duchenne Muscular Dystrophy, sponsored by Genethon. Recruiting at 15 sites in 4 countries. Open to male participants aged 4 Years to 9 Years. Per ClinicalTrials.gov, last updated 2026-02-12.
Sponsored by Genethon · Observational
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.
Study duration from FPFV: Q1 2019 to LPLV: Q4 2026
Primary Ojectives:
Secondary Objectives:
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's planned enrollment of 220 is above the median of 60 across 132 observational studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Genethon is the lead sponsor of 15 studies on the registry; 5 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Body-weight ≤ 95th percentile or the scale ≤ 95th percentile (according to validated scale in force in country site).
Related to the DMD disease:
Able to achieve at inclusion and screening visits:
Ongoing 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:
Exclusion Criteria:
Subject will be excluded from enrolment into the study for any of the following reasons:
Related to the DMD disease severity:
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:
Previously treated with a gene therapy drug for DMD, such as:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NSAA scale
NSAA scale (age appropriate modified North Star Ambulatory Assessment)
Time frame: Screening 36 months
10 Meter Walk/ Run test (10MW/RT)
Time function Test
Time frame: Screening 36 months
Raise from floor (RFF)
Time function test
Time frame: Screening - 36 months
6 Minutes Walk Test (6 MWT)
Motor Function Measurement
Time frame: Inclusion 36 months
Myoset : Myo-grip, -pinch
Motor Function Measurement
Time frame: Inclusion 36 months
Stride velocity 95th centile (SV95c)
Motor Function Measurement with wearable device (SYDE)
Time frame: Inclusion 36 months
Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI)
Muscle Imaging
Time frame: Inclusion 36 months
Pulmonary Function Test (PFT)
Respiratory Function Assessment
Time frame: Inclusion 36 months
ECG - Echocardiography
Cardiac Function Assessment
Time frame: Inclusion 36 months
ACTIVLIM
Patient Reported Outcome
Time frame: Inclusion 36 months
EQ-5D
Questionnaire of Life
Time frame: Inclusion 36 months
Not enrolling at this site.
Started Dec 2019 (actual). Primary completion is estimated for May 2029, 113 months after the start.
Of 52 non-industry observational studies under Muscular Dystrophy, Duchenne, half reached primary completion within 37 months of starting (the median), counting those still running; a quarter within 11 and three quarters within 120. This study's planned 113 months is above that.
Counting every sponsor, half of 63 studies reached it within 40 months; a quarter within 12 and three quarters within 120.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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Muscular Dystrophy, Duchenne →
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