Krabbe Disease Global Patient Registry
Part of paid clinical trials in Buffalo, New York.
- Sponsor
- State University of New York at Buffalo
- Study ID
- NCT02993796
- Status
- Recruiting
Conditions
- Krabbe Disease
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - N/A
- Healthy Volunteers
- Not accepted
Study Details
The purpose of this study is to develop a clinical database of individuals diagnosed with Krabbe disease in order to determine which symptoms herald the onset of clinical disease in the various phenotypes of Krabbe disease; to determine whether level of GALC enzyme activity, or a specific genetic mutation predict the clinical course; and to determine which neurodiagnostic tests predict onset and/or severity of the disease.
Key Dates
- Start date
- Sep 30, 2014
- Status verified
- Feb 2025
- Primary completion
- Sep 30, 2026
- Completion
- Sep 30, 2026
Study Design
- Enrollment
- 60 participants (estimated)
Primary Outcome Measure
Overall Survival [ Time Frame: up to 5 years ]
Central Contacts
- Thomas J. Langan, MD716-888-4732
- Amy Barczykowski716-829-6101
Locations (1)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| State University of New York at Buffalo | Buffalo | New York | 14203 |
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