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Ruxolitinib for Chuvash Polycythemia

Expanded access is currently available for this treatment.
Verified January 2016 by Washington University School of Medicine
Incyte Corporation
Information provided by (Responsible Party):
Washington University School of Medicine Identifier:
First received: November 7, 2012
Last updated: January 6, 2016
Last verified: January 2016
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 Intervention
Erythrocytosis, Familial, 2
Drug: Ruxolitinib

Study Type: Expanded Access     What is Expanded Access?
Official Title: Ruxolitinib for Chuvash Polycythemia

Resource links provided by NLM:

Further study details as provided by Washington University School of Medicine:

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

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

Inclusion Criteria:

  • Diagnosis of Chuvash polycythemia

Exclusion Criteria:

  • Unable to comprehend or unwilling to sign an informed consent form.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01730755

Contact: Karyn Gordon, BSN 314-362-0156
Contact: Jennifer Riggs 314-362-9903

United States, Missouri
Washington University School of Medicine
St. Louis, Missouri, United States, 63110
Contact: Karyn Gordon, BSN    314-362-0156   
Principal Investigator: Stephen Oh, MD, PhD         
Sponsors and Collaborators
Washington University School of Medicine
Incyte Corporation
  More Information

Responsible Party: Washington University School of Medicine Identifier: NCT01730755     History of Changes
Other Study ID Numbers: I-RUX-12-03
Study First Received: November 7, 2012
Last Updated: January 6, 2016

Keywords provided by Washington University School of Medicine:

Additional relevant MeSH terms:
Hematologic Diseases processed this record on May 25, 2017