A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

Limb-girdle Muscular Dystrophy Clinical Trial

Official title:
Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), Type 2C (LGMD2C/R5), and Type 2A (LGMD2A/R1)

Status Recruiting

Clinical Trial Summary

This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy type 2C (LGMD2C/R5), or Limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 3 years after enrollment. Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04475926
Study type	Observational
Source	Sarepta Therapeutics, Inc.
Contact	Sarepta Therapeutics Inc., For Clinical Trial Information, Selec
Phone	1-888-SAREPTA (1-888-727-3782)
Email	SareptAlly@sarepta.com
Status	Recruiting
Phase
Start date	April 22, 2021
Completion date	March 31, 2028

View Details

See also
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Recruiting	`NCT06246513` - A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)	Phase 3
Completed	`NCT00104078` - Study Evaluating MYO-029 in Adult Muscular Dystrophy	Phase 1/Phase 2
Completed	`NCT02759302` - MRI on Persons With Mutations in POMT2 Gene (LGMD2N)	N/A