Ruxolitinib for Chuvash Polycythemia
| Status: | No longer available |
|---|---|
| Conditions: | Hematology |
| Therapuetic Areas: | Hematology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 5/16/2018 |
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.
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.
Study involvement will last for 48 weeks. There will be approximately 11 visits through week
48. Visits may take up to 2-3 hours to complete and occur every 4 weeks for the first 24
weeks, then every 12 weeks until week 48.
During each study visit, any or all of the following procedures may occur:
- List current medications and participant general health
- Obtain blood pressure, body weight, body temperature, respiratory rate and heart rate
- Measure Spleen by examination
- Obtain an abdominal MRI to evaluate spleen and any pre-existing or new blood clots
- Obtain blood samples for safety tests and to monitor kidney/liver function.
- Questionnaires for participant to complete regarding symptoms related to disease.
- Ruxolitinib dosing may be increased after 4 weeks if needed. The dose of the ruxolitinib
may be reduced related to side effects.
48. Visits may take up to 2-3 hours to complete and occur every 4 weeks for the first 24
weeks, then every 12 weeks until week 48.
During each study visit, any or all of the following procedures may occur:
- List current medications and participant general health
- Obtain blood pressure, body weight, body temperature, respiratory rate and heart rate
- Measure Spleen by examination
- Obtain an abdominal MRI to evaluate spleen and any pre-existing or new blood clots
- Obtain blood samples for safety tests and to monitor kidney/liver function.
- Questionnaires for participant to complete regarding symptoms related to disease.
- Ruxolitinib dosing may be increased after 4 weeks if needed. The dose of the ruxolitinib
may be reduced related to side effects.
Inclusion Criteria:
- Diagnosis of Chuvash polycythemia
Exclusion Criteria:
- Unable to comprehend or unwilling to sign an informed consent form.
We found this trial at
1
site
660 S Euclid Ave
Saint Louis, Missouri 63110
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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