NCT03882827 Natural History of Duchenne Muscular Dystrophy
| NCT ID | NCT03882827 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Genethon |
| Condition | Duchenne Muscular Dystrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 220 participants |
| Start Date | 2019-12-19 |
| Primary Completion | 2029-05-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 220 participants in total. It began in 2019-12-19 with a primary completion date of 2029-05-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
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
Contact & Investigator
Francesco MUNTONI, Pr
PRINCIPAL INVESTIGATOR
GOSH LONDON
Frequently Asked Questions
Who can join the NCT03882827 clinical trial?
This trial is open to male participants only, aged 4 Years or older, up to 9 Years, studying Duchenne Muscular Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT03882827 currently recruiting?
Yes, NCT03882827 is actively recruiting participants. Contact the research team at c.griffith@ucl.ac.uk for enrollment information.
Where is the NCT03882827 trial being conducted?
This trial is being conducted at Brussels, Belgium, Leuven, Belgium, Liège, Belgium, Bordeaux, France and 11 additional locations.
Who is sponsoring the NCT03882827 clinical trial?
NCT03882827 is sponsored by Genethon. The principal investigator is Francesco MUNTONI, Pr at GOSH LONDON. The trial plans to enroll 220 participants.