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Ruxolitinib for Chuvash Polycythemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01730755
Expanded Access Status : No longer available
First Posted : November 21, 2012
Last Update Posted : May 14, 2018
Incyte Corporation
Information provided by (Responsible Party):
Washington University School of Medicine

Brief 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.

Condition or disease Intervention/treatment
Erythrocytosis, Familial, 2 Drug: Ruxolitinib

Detailed Description:

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.

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Study Type : Expanded Access
Official Title: Ruxolitinib for Chuvash Polycythemia

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: Ruxolitinib
    Ruxolitinib 10 mg tablets twice daily
    Other Name: Jakafi

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All

Inclusion Criteria:

  • Diagnosis of Chuvash polycythemia

Exclusion Criteria:

  • Unable to comprehend or unwilling to sign an informed consent form.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01730755

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United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
Sponsors and Collaborators
Washington University School of Medicine
Incyte Corporation
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Responsible Party: Washington University School of Medicine Identifier: NCT01730755    
Other Study ID Numbers: I-RUX-12-03
First Posted: November 21, 2012    Key Record Dates
Last Update Posted: May 14, 2018
Last Verified: May 2018
Keywords provided by Washington University School of Medicine:
Additional relevant MeSH terms:
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Hematologic Diseases