Natural History of Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT03882827|
Recruitment Status : Recruiting
First Posted : March 20, 2019
Last Update Posted : March 18, 2021
|Condition or disease|
|Duchenne Muscular Dystrophy|
Study duration from FPFV: Q1 2019 to LPLV: Q3 2023
- 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.
- 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.
|Study Type :||Observational|
|Estimated Enrollment :||100 participants|
|Official Title:||A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 5 to 9 Years|
|Actual Study Start Date :||December 19, 2019|
|Estimated Primary Completion Date :||June 30, 2023|
|Estimated Study Completion Date :||June 30, 2023|
- NSAA scale [ Time Frame: Screening 36 months ]NSAA scale (age appropriate modified North Star Ambulatory Assessment)
- 10 Meter Walk/ Run test (10MW/RT) [ Time Frame: Screening 36 months ]Time function Test
- 6 Minutes Walk Test (6 MWT) [ Time Frame: Screening 36 months ]Motor Function Measurement
- Myoset : Myo-grip, -pinch [ Time Frame: Inclusion 36 months ]Motor Function Measurement
- ACTIMYO [ Time Frame: Inclusion 36 months ]Motor Function Measurement
- Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI) [ Time Frame: Inclusion 36 months ]Muscle Imaging
- Pulmonary Function Test (PFT) [ Time Frame: Inclusion 36 months ]Respiratory Function Assessment
- ECG - Echocardiography [ Time Frame: Inclusion 36 months ]Cardiac Function Assessment
- ACTIVLIM [ Time Frame: Inclusion 36 months ]Patient Reported Outcome
- EQ-5D [ Time Frame: Inclusion 36 months ]Questionnaire of Life
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03882827
|Contact: Francesco MUNTONI, Pr||+44 email@example.com|
|Hôpital Armand Trousseau||Recruiting|
|Paris, France, 75011|
|Strasbourg, France, 67000|
|Great Ormond Street Hospital & University College London Hospital||Not yet recruiting|
|London, United Kingdom, WC1N 1EH|
|Institute of Genetic Medicine||Not yet recruiting|
|Newcastle, United Kingdom|
|Principal Investigator:||Francesco MUNTONI, Pr||GOSH LONDON|