Natural History Study of Patients With VCP-related Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

44 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-03-18

Study Completion Date

2025-09-01

Brief Summary

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

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Detailed Description

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This is a single site, prospective natural history study to evaluate the utility of strength, functional, and patient reported outcomes in quantifying disease progression over 1 year in patients with mutations in the VCP gene.

Patients will complete a remote and onsite visits at baseline, a remote visit at 6 months, and then a final remote and onsite visit at 12 months. All strength, functional, and patient reported measures will be completed at each visit to assess reliability, consistency of results across testing environments, and sensitivity to change over time.

Additionally, patients may opt in to provide blood, serum, and urine samples for biobanking.

Conditions

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IBMPFD

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No