Defining Clinical Endpoints in LGMD
Research type
Research Study
Full title
Defining Clinical Endpoints in LGMD
IRAS ID
271534
Contact name
Jordi Diaz-Manera
Contact email
Sponsor organisation
Virginia Commonwealth University
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 5 months, 31 days
Research summary
The study will enrol participants from a range of limb girdle muscular dystrophies to perform a 12 months longitudinal study. There are proposed gene replacements therapies in pre-clinical testing for at least three of the conditions being assessed meaning that there is an urgent need for natural history data. The study will use standard clinical outcome assessments for these conditions to hasten therapeutic development. Participants will be aged between 4-65 at the time of enrolment and must be ambulant. As the study is observational data collected will show the progression over 12 months according to a range of standard outcome measures. The study will also use biomarkers so blood and urine samples will be taken.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
22/NW/0008
Date of REC Opinion
9 Mar 2022
REC opinion
Further Information Favourable Opinion