Ruxolitinib for Chuvash Polycythemia

Expanded access is currently available for this treatment.
Verified November 2012 by Washington University School of Medicine
Sponsor:
Collaborator:
Incyte Corporation
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT01730755
First received: November 7, 2012
Last updated: November 15, 2012
Last verified: November 2012

November 7, 2012
November 15, 2012
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Complete list of historical versions of study NCT01730755 on ClinicalTrials.gov Archive Site
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Ruxolitinib for Chuvash Polycythemia
Ruxolitinib for Chuvash Polycythemia

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 regulates the activity of JAK2. 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.
Expanded Access
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Chuvash Polycythemia
Drug: Ruxolitinib
Ruxolitinib 10 mg tablets twice daily
Other Name: Jakafi
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Available
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Inclusion Criteria:

  • Diagnosis of Chuvash polycythemia

Exclusion Criteria:

  • Unable to comprehend or unwilling to sign an informed consent form.
Both
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Contact: Stephen Oh, MD, PhD 314-362-8846 stoh@dom.wustl.edu
United States
 
NCT01730755
I-RUX-12-03
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Washington University School of Medicine
Washington University School of Medicine
Incyte Corporation
Not Provided
Washington University School of Medicine
November 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP