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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
History of Changes
  Purpose

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.


Condition Intervention
Chuvash Polycythemia
 Drug: Ruxolitinib

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

Resource links provided by NLM:

U.S. FDA Resources

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.
  Eligibility

Genders Eligible for Study:   Both
Criteria

Inclusion Criteria:

  • Diagnosis of Chuvash polycythemia

Exclusion Criteria:

  • Unable to comprehend or unwilling to sign an informed consent form.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01730755

Contacts
Contact: Stephen Oh, MD, PhD 314-362-8846 stoh@dom.wustl.edu

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

Additional Information:
Prescribing information  This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT01730755     History of Changes
Other Study ID Numbers: I-RUX-12-03
Study First Received: November 7, 2012
Last Updated: November 15, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Washington University School of Medicine:
Polycythemia

Additional relevant MeSH terms:
Polycythemia
Hematologic Diseases

ClinicalTrials.gov processed this record on May 23, 2013