Status:
ACTIVE_NOT_RECRUITING
Natural History Study of Patients With VCP-related Disease
Lead Sponsor:
Nationwide Children's Hospital
Conditions:
IBMPFD
Eligibility:
All Genders
18+ years
Brief Summary
A natural history study to understand the expected progression of disease in patients with confirmed mutations in the VCP gene over 1 year.
Detailed Description
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 ...
Eligibility Criteria
Inclusion
- Genetically confirmed mutation in the VCP gene
- Age ≥18 years
- Willing and able to provide informed consent and follow all study procedures
Exclusion
- 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.
Key Trial Info
Start Date :
March 18 2021
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
September 1 2025
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT04823143
Start Date
March 18 2021
End Date
September 1 2025
Last Update
February 11 2025
Active Locations (1)
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1
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205