Natural History of Duchenne Muscular Dystrophy

Last updated: September 5, 2022
Sponsor: Genethon
Overall Status: Active - Recruiting

Phase

N/A

Condition

Muscular Dystrophy

Treatment

N/A

Clinical Study ID

NCT03882827
GNT-014-MDYF
  • Ages 5-9
  • Male

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 5 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Male
  2. 5 to 9 years old inclusive
  3. Body-Weight < or = 75th percentile of BMI body-mass index scale (according tovalidated scale in force in the country site)
  4. Diagnosis of DMD based upon Gene testing positive with detailed genotyping
  5. Able to achieve:
  • NSAA (North Star Ambulatory Assessment) scale > or =18 (with a maximum of 2points difference between inclusion and screening visits) and/or:
  • Gowers test < or =7 sec
  • 6 Minute Walk Test (6MWT) > or = 350 meters at screening visit (M1) and atinclusion visit (M0) with the distance being 20% of each other
  1. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according tostandard of care in the previous 3 months
  2. Signed informed consent by at least one parent(s) or both parents or legal guardianrepresentative(s), when applicable and according to the country regulation
  3. Affiliated Beneficiary of the National Health Care scheme

Exclusion

Exclusion Criteria: 9. Cardiomyopathy based on physical cardiological examination and echocardiography withLeft Ventricular 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 mayinterfere with DMD natural evolution and or evaluation of outcomes designed to assessDMD Natural History 12. Muscle testing: inability to cooperate with 13. Nuclear Magnetic Resonance Imaging (NMRI): metal implants in regions of interest forthe study 14. Unwilling and/or unable to comply with all the study protocol requirements and orprocedures 15. Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months (or IMP washout period) prior to the screening visit of thestudy 16. Concomitant participation to any other clinical trial

Study Design

Total Participants: 100
Study Start date:
December 19, 2019
Estimated Completion Date:
June 30, 2023

Study Description

Study duration from FPFV: Q1 2019 to LPLV: Q3 2023

Primary Ojectives:

  • To assess the natural disease course using standardized and disease appropriate evaluations over a period of 6 to 36 months in a cohort of young male subjects aged from 5 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD).

  • To record a baseline period prior to the setup of an AAV gene therapy dose escalation phase I/II First in Man 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.

Connect with a study center

  • University Hospital of Bordeaux

    Bordeaux, 33076
    France

    Active - Recruiting

  • Brest University Hospital Centre

    Brest, 29609
    France

    Active - Recruiting

  • Hopital Femme Mere Enfant

    Bron, 69677
    France

    Active - Recruiting

  • CHU Lille

    Lille, 59000
    France

    Active - Recruiting

  • Hopital la Timone Enfants

    Marseille, 13385
    France

    Active - Recruiting

  • Hôpital Armand Trousseau

    Paris, 75011
    France

    Active - Recruiting

  • Hôpital Hautepierre

    Strasbourg, 67000
    France

    Active - Recruiting

  • Great Ormond Street Hospital & University College London Hospital

    London, WC1N 1EH
    United Kingdom

    Active - Recruiting

  • Institute of Genetic Medicine

    Newcastle,
    United Kingdom

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.