Clinical Trials in Spain / NCT03882827
Recruiting
Observational
Natural History of Duchenne Muscular Dystrophy
NCT03882827 · tracked via the Priya Life Science Spain tracker
Condition(s) studied
Duchenne Muscular Dystrophy
Study 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
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
Primary outcome measure(s)
- NSAA scale — Screening 36 months
NSAA scale (age appropriate modified North Star Ambulatory Assessment) - 10 Meter Walk/ Run test (10MW/RT) — Screening 36 months
Time function Test - Raise from floor (RFF) — Screening - 36 months
Time function test - 6 Minutes Walk Test (6 MWT) — Inclusion 36 months
Motor Function Measurement - Myoset : Myo-grip, -pinch — Inclusion 36 months
Motor Function Measurement - Stride velocity 95th centile (SV95c) — Inclusion 36 months
Motor Function Measurement with wearable device (SYDE) - Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI) — Inclusion 36 months
Muscle Imaging - Pulmonary Function Test (PFT) — Inclusion 36 months
Respiratory Function Assessment - ECG - Echocardiography — Inclusion 36 months
Cardiac Function Assessment - ACTIVLIM — Inclusion 36 months
Patient Reported Outcome - EQ-5D — Inclusion 36 months
Questionnaire of Life
Trial sites (15)
| Facility | City | Region | Status |
|---|---|---|---|
| Centre Hospitalier Universitaire Brugmann | Brussels | Belgium | Recruiting |
| UZ Leuven | Leuven | Belgium | Recruiting |
| CHR Hôpital de la Citadelle | Liège | Belgium | Not Yet Recruiting |
| University Hospital of Bordeaux | Bordeaux | France | Recruiting |
| Brest University Hospital Centre | Brest | France | Recruiting |
| Hopital Femme Mere Enfant | Bron | France | Recruiting |
| CHU Lille | Lille | France | Recruiting |
| Hopital la Timone Enfants | Marseille | France | Recruiting |
| Centre Hospitalier Universitaire - Hôpital Gui de Chauliac | Montpellier | France | Not Yet Recruiting |
| Hôpital Armand Trousseau | Paris | France | Recruiting |
| Hôpital Hautepierre | Strasbourg | France | Recruiting |
| Hospital Sant Joan de Deu Esplugues de Llobregat | Esplugues de Llobregat | Spain | Recruiting |
| Hospital La Fe de Valencia | Valencia | Spain | Active Not Recruiting |
| Great Ormond Street Hospital & University College London Hospital | London | United Kingdom | Recruiting |
| Institute of Genetic Medicine | Newcastle | United Kingdom | Recruiting |
More Genethon trials in Spain
Other trials for the same condition
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Active, not recruiting
Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies.
Enrolling by invitation
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03882827 on ClinicalTrials.gov ↗ ← All trials in Spain