At a glance
- What the study gets you
- Health checks and monitoring — no treatment given
- Type of study
- Observational (no treatment given)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Not specified by the sponsor
- How long the study runs
- Study runs about 117 months (dates as stated)
- About the drug or intervention
- Not specified by the sponsor
- Patient visit burden
- Not specified by the sponsor
In plain English
This study looks at the natural history of Duchenne muscular dystrophy (DMD), meaning it tracks how the condition develops over time. It is sponsored by Genethon and involves boys aged 4 to 9 with DMD. The study does not test a new medicine.
Who can take part
- Boys aged 4 to 9 years old
- Weight or body mass index (BMI) at or below the 95th percentile
- Diagnosis of DMD confirmed by genetic testing with detailed results
- Able to meet certain movement tests at the screening and inclusion visits, including a North Star Ambulatory Assessment (NSAA) score above 18 (or 16 or more if aged 4 to under 5), a Gowers test of 7 seconds or less, and/or a 6-minute walk test of at least 350 metres
- Already taking corticosteroid medicine, or starting it as part of standard care before the screening visit
- Consent signed by a parent, parents, or legal guardian as required by local rules
- Member of, or covered by, a health care scheme as required by local rules
Who may not be able to
- Heart problems (cardiomyopathy) with a heart pumping measure (left ventricular ejection fraction) below 55%
- Needing breathing support during the day and/or night
- Other health conditions or past/planned surgery that could affect how DMD develops or how the study results are measured
- Unable to cooperate with muscle testing
- Metal implants in areas the study scans with MRI (magnetic resonance imaging)
- Unwilling or unable to follow all study requirements and procedures
- Took part in another trial of an investigational medicine within the last 3 months, or within the washout period if longer
- Previously treated with gene therapy for DMD, or certain exon-skipping drugs too close to the screening visit
- Currently taking part in any other interventional clinical trial
What taking part involves
- • No study medicine is given; the study observes the natural course of Duchenne muscular dystrophy
- • Movement tests such as the NSAA, the Gowers test, and a 6-minute walk test
- • Heart checks including an echocardiography (heart ultrasound)
- • MRI scans of the muscles
Time commitment: Not stated — ask the trial team about how many visits there are, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Observing health over time
- Ages
- 4 Years to 9 Years
- Who
- Male
- Number of participants
- 220
- Started
- 2019-12-19
- Last checked
- 2026-02
Plain English Summary
What is this study?
- • Testing a new treatment for duchenne muscular dystrophy
- • Clinical study - 220 participants
- • 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
Who can take part?
- • Ages 4 Years to 9 Years
- • Diagnosed with duchenne muscular dystrophy
- • Male only
Where?
- • London - Great Ormond Street Hospital & University College London Hospital
- • Newcastle - Institute of Genetic Medicine
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
More detail
Study duration from FPFV: Q1 2019 to LPLV: Q4 2026 Primary Ojectives: * To assess the natural disease course using standardized and disease appropriate evaluations in a cohort of young male subjects aged from 4 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD). * To record a baseline period prior to rolling over into a gene therapy phase I/II/III clinical study. Secondary Objectives: * To identify clinical, imaging and/or laboratory parameters that could be predictive indicators of the disease course in DMD, within the selected range of age. * To identify the best outcome measure(s) for further clinical trial assessments.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 4 Years - 9 Years
- Who can join: Male only
What the study is looking for
- ✓4 to 9 years old inclusive
- ✓Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country...
- ✓Related to the DMD disease:
- ✓Diagnosis of DMD based upon documented gene testing with detailed genotyping
- ✓Able to achieve at inclusion and screening visits:
Who cannot take part
- ✗Subject will be excluded from enrolment into the study for any of the following reasons:
- ✗Related to the DMD disease severity:
- ✗Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson...
- ✗Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
- ✗Any co-morbidity (ies) and or previous or planned surgical event(s) which may interfere with DMD natural evolution...
See the full criteria
Where Is This Study? (2 UK sites)
Great Ormond Street Hospital & University College London Hospital
London WC1N 1EH, United Kingdom
Main Email: Research.Governance@gosh.nhs.uk
Research.Governance@gosh.nhs.uk0207 905 2700Institute of Genetic Medicine
Newcastle, United Kingdom
How to Get in Touch
Francesco MUNTONI, Pr
Sponsor contactCONTACT