Recruiting

Observational Study

Sponsor:

Virginia Commonwealth University

Code:

NCT05989620

Conditions

LGMD1B

LGMD1C

LGMD1D

LGMD1E

LGMD1F

Eligibility Criteria

Sex: All

Age: 6 - 50

Healthy Volunteers: Not accepted

Study Details

Brief 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).

Conditions

LGMD1B

LGMD1C

LGMD1D

LGMD1E

LGMD1F

Study ID

NCT05989620

Start date

Oct 18, 2023

Status verified date

Nov, 2025

Completion date

May 1, 2029

Anticipated

Primary completion date

Oct 1, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 50

Healthy Volunteers: Not accepted

Inclusion 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

Exclusion Criteria:

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

Study Design

Enrollment

1000 participants

Anticipated

Interventions and Outcome Measures

Arms

LGMD, DM2, LOPD

Primary outcome measure

  • To explore the suitability and feasibility of the North Star Assessment for LGMD (NSAD) in the muscular dystrophies [ Time Frame: Baseline to 24 months ]

Central Contacts and Locations

Central contacts

Jennifer Raymond

804-828-6318Jennifer.Raymond@vcuhealth.org

Ruby Langeslay

Ruby.Langeslay@vcuhealth.org

Locations

Virginia Commonwealth University

Recruiting

Richmond, Virginia, United States, 23298

Contacts

Levi Headrick

levi.headrick@vcuhealth.org

Principal Investigator:

Nicholas Johnson, MD

More Information

Sponsor

Virginia Commonwealth University

Last update posted

Nov 18, 2025

Last verified

Nov, 2025

Keywords

  • Muscular Dystrophy
  • Myotonic Dystrophy
  • Limb Girdle Muscular Dystrophy
  • LGMD
  • LOPD
  • Late Onset Pompe Disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Virginia Commonwealth University on 2025-11-18.