Long-Term Development of Muscular Dystrophy Outcome Assessments

Description

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).

Conditions

LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y

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Study Overview

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Study Details

Study overview

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).

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

Long-Term Development of Muscular Dystrophy Outcome Assessments

Condition
LGMD1B
Intervention / Treatment

-

Contacts and Locations

Richmond

Virginia Commonwealth University, Richmond, Virginia, United States, 23298

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Age between 6-50 years at enrollment
  • 2. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness)
  • 3. Genetic confirmation of a LGMD, DM2, or LOPD
  • 4. FVC above 30% of predicted
  • 1. 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
  • 2. Participation in a clinical trial receiving an investigational product

Ages Eligible for Study

6 Years to 50 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Virginia Commonwealth University,

Nicholas Johnson, MD, PRINCIPAL_INVESTIGATOR, Virginia Commonwealth University

Study Record Dates

2029-05-01