RecruitingNCT05989620

Long-Term Development of Muscular Dystrophy Outcome Assessments

Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)


Sponsor

Virginia Commonwealth University

Enrollment

1,000 participants

Start Date

Oct 18, 2023

Study Type

OBSERVATIONAL

Conditions

Summary

This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).


Eligibility

Min Age: 6 YearsMax Age: 50 Years

Inclusion Criteria4

  • Age between 6-50 years at enrollment
  • Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness)
  • Genetic confirmation of a LGMD, DM2, or LOPD
  • FVC above 30% of predicted

Exclusion Criteria2

  • Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator
  • Participation in a clinical trial receiving an investigational product

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Locations(1)

Virginia Commonwealth University

Richmond, Virginia, United States

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NCT05989620


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