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 ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

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

View Details

See also
Status	Clinical Trial	Phase
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Recruiting	`NCT05230459` - A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)	Phase 1/Phase 2
Completed	`NCT02841267` - A Trial of PF-06252616 in Ambulatory Participants With LGMD2I	Phase 1/Phase 2
Active, not recruiting	`NCT04800874` - Study of BBP-418 in Patients With LGMD2I	Phase 2