Scientists track rare muscle disease progression in 44 patients

NCT ID NCT04823143

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

Summary

This study followed 44 adults with a confirmed VCP gene mutation to learn how their disease (IBMPFD) naturally progresses over one year. Participants completed walking tests, strength assessments, and surveys about daily function and quality of life. The goal was to gather data to help design future treatment trials.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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

Contacts and locations

Locations

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States