Natural History of Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT03882827|
Recruitment Status : Not yet recruiting
First Posted : March 20, 2019
Last Update Posted : May 14, 2019
|Condition or disease|
|Duchenne Muscular Dystrophy|
Study duration from FPFV: Q1 2019 to LPLV: Q3 2021
- To assess the natural disease course using standardized and disease appropriate evaluations over a period of 6 to 24 months in a cohort of young male subjects aged from 4 to 6 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 :||30 participants|
|Official Title:||A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 6 Years|
|Estimated Study Start Date :||June 30, 2019|
|Estimated Primary Completion Date :||April 30, 2021|
|Estimated Study Completion Date :||October 30, 2021|
- NSAA scale [ Time Frame: Screening 24 months ]NSAA scale (age appropriate modified North Star Ambulatory Assessment)
- 10 Meter Walk/ Run test (10MW/RT) [ Time Frame: Screening 24 months ]Time function Test
- 6 Minutes Walk Test (6 MWT) [ Time Frame: Screening 24 months ]Motor Function Measurement
- Myoset : Myo-grip, -pinch [ Time Frame: Inclusion 24 months ]Motor Function Measurement
- ACTIMYO [ Time Frame: Inclusion 24 months ]Motor Function Measurement
- Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI) [ Time Frame: Inclusion 24 months ]Muscle Imaging
- Pulmonary Function Test (PFT) [ Time Frame: Inclusion 24 months ]Respiratory Function Assessment
- ECG - Echocardiography [ Time Frame: Inclusion 24 months ]Cardiac Function Assessment
- ACTIVLIM [ Time Frame: Inclusion 24 months ]Patient Reported Outcome
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 firstname.lastname@example.org|
|Hôpital Armand Trousseau||Not yet recruiting|
|Paris, France, 75011|
|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|