Natural History of Duchenne Muscular Dystrophy

Status Recruiting

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT03882827
Study type	Observational
Source	Genethon
Contact	Francesco MUNTONI, Pr
Phone	+44 02079052602
Email	c.griffith@ucl.ac.uk
Status	Recruiting
Phase
Start date	December 19, 2019
Completion date	June 30, 2023

View Details

See also
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