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Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

NCT03981289 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 116

Last updated 2026-02-27

No results posted yet for this study

Summary

Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

Conditions

  • Limb Girdle Muscular Dystrophy
  • Muscular Dystrophies

Sponsors & Collaborators

  • Newcastle University

    collaborator OTHER

  • Nationwide Children's Hospital

    collaborator OTHER

  • Washington University School of Medicine

    collaborator OTHER

  • University of Iowa

    collaborator OTHER

  • University of Minnesota

    collaborator OTHER

  • University of California, Irvine

    collaborator OTHER

  • Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

    collaborator OTHER

  • University of Colorado, Denver

    collaborator OTHER

  • University of Kansas Medical Center

    collaborator OTHER

  • Virginia Commonwealth University

    lead OTHER

Principal Investigators

  • Nicholas Johnson, MD · Virginia Commonwealth University

Eligibility

Min Age
4 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-06-14
Primary Completion
2025-06-30
Completion
2025-06-30

Countries

  • United States
  • 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 NCT03981289 on ClinicalTrials.gov