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New study maps key tests for LGMD to speed up drug development

NCT ID NCT03981289

Knowledge-focused Sponsor: Virginia Commonwealth University Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study involved 116 people with Limb Girdle Muscular Dystrophy (LGMD), a group of rare muscle-weakening disorders. Researchers measured how well participants could walk, move their arms, and breathe, and asked about their daily activities and overall health. The goal was to identify the best tests to track disease progression, which will help design future clinical trials for new treatments.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

116 people

The number who actually took part.

Started

Jun 2019

Finished

Jun 2025

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

GRASP-LGMD covers a broad geographical distribution and we expect the race and ethnicity to match that of the United States. However, past experience in both the clinical and research LGMD populations shows that some minority subgroups are under-represented in research studies. To assist with recruitment of persons from diverse backgrounds we will utilize the Community and Special Populations Engagement Personnel supported through the CTSA networks to reach out to under-represented communities through a variety of local techniques which could include direct outreach, advertising with local advocacy groups, organizations, or newsletters, and local advertising using a variety of media which could include Social Media (e.g. Facebook, Twitter), radio, and newspapers.

Ages

4 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria - Arm 1: * Age between 4-65 at enrollment * Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity) * A genetically or functionally confirmed mutation in ANO5, CAPN3, DYSF, DNAJB6 or SGCA-G. * Willing and able to give informed consent and follow all study procedures and requirements Inclusion Criteria - Arm 2: * Age between 4-65 at enrollment * Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity) * a genetically confirmed mutation in SGCA-G * Willing and able to give informed consent and follow all study procedures and requirements Exclusion Criteria - Arm 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. * History of a bleeding disorder, platelet count \<50,000, current use of an anticoagulant. * Positive pregnancy test at time any timepoint during the trial. Exclusion Criteria - Arm 2: * 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. * History of a bleeding disorder, platelet count \<50,000, current use of an anticoagulant * Positive pregnancy test at time any timepoint during the trial.

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Conditions

The condition(s) this trial relates to.

autosomal dominant limb-girdle muscular dystrophy type 1D (DNAJB6) limb-girdle muscular dystrophy muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Limb girdle muscular dystrophy Muscular dystrophies

Contacts and locations

Locations

  • John Walton Muscular Dystrophy Research Centre (Newcastle Upon Tyne)

    Newcastle, United Kingdom

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • The University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • University of California Irvine

    Irvine, California, 92697, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.