CompletedNot Applicable

Natural History Study of Patients With VCP-related Disease

United States44 participantsStarted 2021-03-18

Plain-language summary

A natural history study to understand the expected progression of disease in patients with confirmed mutations in the VCP gene over 1 year.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Genetically confirmed mutation in the VCP gene * Age ≥18 years * Willing and able to provide informed consent and follow all study procedures Exclusion Criteria: * Participation in an interventional clinical trial * Any concomitant illness or comorbid condition that would interfere with a patient's ability to complete study procedures safely at the discretion of the site PI.

What they're measuring

100 meter timed test

Timeframe: 12 months

North Star assessment for limb girdle type dystrophies (NSAD)

Timeframe: 12 months

Timed Up and Go

Timeframe: 12 months

4 stair climb

Timeframe: 12 months

Performance of Upper Limb (PUL) 2.0

Timeframe: 12 months

9 hole peg test

Timeframe: 12 months

Forced Vital Capacity

Timeframe: 12 months

PROMIS Global Health scale

Timeframe: 12 months

Neuro-QOL Upper Extremity Function

Timeframe: 12 months

Trial details

NCT IDNCT04823143

SponsorNationwide Children's Hospital

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2025-08-31

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