LGMD R1 Natural History Study

(GRASP-01-003 Trial)

Recruiting at 11 trial locations

Overseen By Jennifer Raymond

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: Virginia Commonwealth University

Disqualifiers: Non-ambulatory, Pregnancy, Dominant CAPN3, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a 24-month, observational study of 100 participants with Limb Girdle Muscular Dystrophy type R1, also known as CAPN3.

Who Is on the Research Team?

Nicholas Johnson, MD

Principal Investigator

Virginia Commonwealth University

Are You a Good Fit for This Trial?

Inclusion Criteria

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).

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Baseline Assessment

Initial assessments including MR scans and baseline measurements

2 days

2 visits (in-person)

Observational Study

Participants undergo observational assessments including MR scans and various functional tests

24 months

4 visits (in-person) at Baseline Day 1, Baseline Day 2, Month 12, and Month 24

Follow-up

Participants are monitored for safety and effectiveness after the observational period

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Observational Study

Who Is Running the Clinical Trial?

Virginia Commonwealth University

Lead Sponsor

Trials

732

Recruited

22,900,000+

University of Minnesota

Collaborator

Trials

1,459

Recruited

1,623,000+

Community Health Clinic

Collaborator

Trials

1

Recruited

100+

Amsterdam/Leiden University Medical Center

Collaborator

Trials

1

Recruited

100+

Newcastle University

Collaborator

Trials

172

Recruited

14,120,000+

University of Kansas Medical Center

Collaborator

Trials

527

Recruited

181,000+

Indiana CHC (Community Health Clinic)

Collaborator

Trials

1

Recruited

100+

University of California, Irvine

Collaborator

Trials

580

Recruited

4,943,000+

University of Kansas

Collaborator

Trials

157

Recruited

332,000+

University of Colorado, Denver

Collaborator

Trials

1,842

Recruited

3,028,000+