NCT03882827

Natural History of Duchenne Muscular Dystrophy

A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 220 (estimated)
• Sponsor: Genethon · Academic / Other
• Sex: Male
• Age: 4 Years – 9 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Study duration from FPFV: Q1 2019 to LPLV: Q4 2026 Primary Ojectives: * To assess the natural disease course using standardized and disease appropriate evaluations in a cohort of young male subjects aged from 4 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD). * To record a baseline period prior to rolling over into a gene therapy phase I/II/III 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.

Conditions

• Duchenne Muscular Dystrophy

Timeline

• Start date: 2019-12-19
• Primary completion: 2029-05-30
• Completion: 2029-09-30
• First posted: 2019-03-20
• Last updated: 2026-02-12

Locations

15 sites across 4 countries: Belgium, France, Spain, United Kingdom