Ruxolitinib for Chuvash Polycythemia
NCT01730755 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2018-05-14
Summary
Chuvash polycythemia (CP) is a rare form of congenital polycythemia caused by mutations in the VHL gene. Currently, there are no therapies that have proven effective for CP. Recent studies have demonstrated that VHL (von Hippel-Lindau tumor suppressor) regulates the activity of JAK2 (Janus kinase 2). In mouse models, inhibition of JAK2 reverses the CP phenotype. Therefore, the investigators hypothesize that JAK2 inhibition may have significant clinical benefits for CP patients.
Conditions
- Erythrocytosis, Familial, 2
Interventions
- DRUG
-
Ruxolitinib 10 mg tablets twice daily
Sponsors & Collaborators
- collaborator INDUSTRY
-
Washington University School of Medicine
lead OTHER
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
Countries
- United States
Study Locations
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