Long-Term Development of Muscular Dystrophy Outcome Assessments

ConditionsLGMD1B 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

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age6-50

SponsorVirginia Commonwealth University

About this trial

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 criteria

Qualifiers

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

Disqualifiers

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

Trial design

Treatments tested in this trial

Not listed

Trial groups

1,000 Participants

are grouped into 1 trial group

Sponsors and collaborators

Virginia Commonwealth University

Lead sponsor

Muscular Dystrophy Association

Collaborator

Source ClinicalTrials.gov