GRASP 01-003 CALPAIN
Research type
Research Study
Full title
Trial Readiness and Endpoint Assessment in LGMD R1 (TREATing LGMDR1)
IRAS ID
352485
Contact name
Jordi Diaz-Manera
Contact email
Sponsor organisation
Virginia Commonwealth University
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 9 months, 22 days
Research summary
This study is a project created to capture natural history data on patients with Limb Girdle Muscular Dystrophy type R1 (LGMDR1, also LGMD2A) to address the gaps in therapeutic development. Currently, there is no approved treatment for this disease group and the results of this study are expected to be used by academics and industry to plan clinical trials in LDMGR1.
This study aims will focus on three key areas:Evaluating the North Star Assessment for LGMD (NSAD) to confirm its reliability for use in LGMDR1 drug trials, including understanding how much scores vary to help design better trials.
Developing standardized training for research teams working across multiple sites to ensure consistent and high-quality data collection.
Testing muscle MRI to see if it can reliably measure changes in muscle fat as an early sign of treatment effects for LGMDR1.
The findings from this study will help researchers and pharmaceutical companies plan future clinical trials for LGMDR1 treatments more effectively.REC name
Wales REC 5
REC reference
25/WA/0021
Date of REC Opinion
21 Mar 2025
REC opinion
Further Information Favourable Opinion