GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1
Status: Recruiting
Location: See all (12) locations...
Study Type: Observational
SUMMARY
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 50
Healthy Volunteers: f
View:
• Age between 12-50 at enrollment
• Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with LGMDR1)
• Genetic confirmation of LGMDR1 (presence of homozygous or compound heterozygous pathogenic mutations in CAPN3).
• Must be able to provide written informed consent and be willing and able to comply with all study requirements. Note: Adult participants must be able to provide consent themselves. Legally authorized representatives are not permitted to consent on behalf of adult participants.
Locations
United States
California
University of California, Irvine
RECRUITING
Orange
Colorado
University of Colorado Anschutz Medical Campus
RECRUITING
Aurora
Florida
University of Florida
NOT_YET_RECRUITING
Gainesville
Iowa
University of Iowa Hospitals and Clinics
RECRUITING
Iowa City
Indiana
The Community Health Clinic, Inc.
RECRUITING
Shipshewana
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Minnesota
University of Minnesota, Department of Neurology
RECRUITING
Minneapolis
Missouri
Washington University School of Medicine
RECRUITING
St Louis
Ohio
Nationwide Children's Hospital
RECRUITING
Columbus
Virginia
Virginia Commonwealth University
RECRUITING
Richmond
Other Locations
Netherlands
Leiden University Medical Center
NOT_YET_RECRUITING
Leiden
United Kingdom
Newcastle University
NOT_YET_RECRUITING
Newcastle Upon Tyne
Contact Information
Primary
Ruby Langeslay
ruby.langeslay@vcuhealth.org
804-828-6318
Backup
Jennifer Raymond
jennifer.raymond@vcuhealth.org
Time Frame
Start Date: 2024-01-31
Estimated Completion Date: 2028-08
Participants
Target number of participants: 100
Treatments
LGMD Type R1/LGMD2A/CAPN3
No intervention will be administered.
Related Therapeutic Areas
Sponsors
Collaborators: Indiana CHC (Community Health Clinic), Washington University School of Medicine, Nationwide Children's Hospital, Newcastle University, University of Iowa, University of Florida, University of Kansas Medical Center, University of California, Irvine, University of Colorado, Denver, University of Minnesota
Leads: Virginia Commonwealth University