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LGMD R1 Natural History Study

NCT05618080 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 100

Last updated 2025-06-08

No results posted yet for this study

Summary

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

Conditions

  • Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A
  • Limb Girdle Muscular Dystrophy
  • Limb Girdle Muscular Dystrophy Type R1
  • LGMD2A

Sponsors & Collaborators

  • Nationwide Children's Hospital

    collaborator OTHER

  • Washington University School of Medicine

    collaborator OTHER

  • University of Iowa

    collaborator OTHER

  • University of Florida

    collaborator OTHER

  • University of Minnesota

    collaborator OTHER

  • Newcastle University

    collaborator OTHER

  • University of Kansas Medical Center

    collaborator OTHER

  • University of Colorado, Denver

    collaborator OTHER

  • Indiana CHC (Community Health Clinic)

    collaborator UNKNOWN

  • University of California, Irvine

    collaborator OTHER

  • Virginia Commonwealth University

    lead OTHER

Principal Investigators

  • Nicholas Johnson, MD · Virginia Commonwealth University

Eligibility

Min Age
12 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-01-31
Primary Completion
2028-05-31
Completion
2028-08-31

Countries

  • United States
  • Netherlands
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05618080 on ClinicalTrials.gov