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Defining Endpoints in Becker Muscular Dystrophy — GRASP-01-002  

Muscular Dystrophies Clinical Trial

Official title:
Defining Endpoints in Becker Muscular Dystrophy

Clinical Trial Summary

This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

Clinical Trial Description

Becker Muscular Dystrophy (BMD) is most frequently due to in-frame mutations in the dystrophin gene that are associated with reduced levels of frequently shortened dystrophin, though other mutations may be related to the Becker phenotype. There is wide variation in the age of onset and degree of progression, ranging from childhood to late adulthood. The more severe form of dystrophinopathy, Duchenne muscular dystrophy, has a more characteristic rate of progression and overall natural history. The wide variation in severity of progression has led to challenges in the design and conduct of approaching therapeutic trials. There is a need for a more rigorous natural history study to assist in the design of these promising therapeutic trials. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05257473
Study type Observational
Source Virginia Commonwealth University
Contact Ruby Langeslay
Phone 804-828-8481
Email ruby.langeslay@vcuhealth.org
Status Recruiting
Phase
Start date April 13, 2022
Completion date May 2026
View Details
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