Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I

LGMD2I Clinical Trial

Official title: Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Limb-Girdle Muscular Dystrophy 2I

Status Active, not recruiting

Clinical Trial Summary

Prospective, longitudinal, interventional, single-group, multicenter natural history study to better know the LGMD2I disease physiopathology. The duration of participation for each patient will be up to 24 months.

Clinical Trial Description

Study duration Duration from First visit of first patient (FPFV) to Last visit of last patient (LPLV) : 3 years

Study objectives Primary objective:

To characterize the disease course in Limb-Girdle Muscular Dystrophy 2I (LGMD2I) patients using standardized and disease appropriate evaluations.

Secondary objectives:

To identify clinical, imaging and/or laboratory parameters that are indicators of the disease course in LGMD2I To identify the best outcome measure for further therapeutics approaches ;

Related Conditions & MeSH terms

• LGMD2I
• Muscular Dystrophies

• NCT number: NCT03842878
• Study type: Observational
• Source: Genethon
• Status: Active, not recruiting
• Start date: February 19, 2020
• Completion date: December 30, 2023