Actively Recruiting
LGMD R1 Natural History Study
Led by Virginia Commonwealth University · Updated on 2025-06-08
100
Participants Needed
12
Research Sites
234 weeks
Total Duration
On this page
Sponsors
V
Virginia Commonwealth University
Lead Sponsor
N
Nationwide Children's Hospital
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.
CONDITIONS
Official Title
LGMD R1 Natural History Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 12 and 50 years at enrollment
- Clinically affected with weakness consistent with LGMDR1
- Genetic confirmation of LGMDR1 with homozygous or compound heterozygous pathogenic CAPN3 mutations
- Able to provide written informed consent and comply with study requirements (adults must consent themselves)
You will not qualify if you...
- Contraindications to MRI or MRS such as non-MR compatible implants or severe claustrophobia
- Non-ambulatory and unable to walk 10 meters without assistive devices (ankle foot orthotics excluded)
- Positive pregnancy test at any time during the trial
- Dominantly inherited CAPN3 mutations (LGMDD4)
- Any illness interfering with safe testing or interpretation of results as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 12 locations
1
University of California, Irvine
Orange, California, United States, 92868
Actively Recruiting
2
University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
3
University of Florida
Gainesville, Florida, United States, 32610
Not Yet Recruiting
4
The Community Health Clinic, Inc.
Shipshewana, Indiana, United States, 46565
Actively Recruiting
5
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
Actively Recruiting
6
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Actively Recruiting
7
University of Minnesota, Department of Neurology
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
8
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
9
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
10
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Actively Recruiting
11
Leiden University Medical Center
Leiden, Netherlands
Not Yet Recruiting
12
Newcastle University
Newcastle upon Tyne, United Kingdom
Not Yet Recruiting
Research Team
R
Ruby Langeslay
CONTACT
J
Jennifer Raymond
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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