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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
Sponsor:
ClinicalTrials.gov Identifier:
NCT01730755
First Posted: November 21, 2012
Last Update Posted: January 8, 2016
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Incyte Corporation
Information provided by (Responsible Party):
Washington University School of Medicine
November 7, 2012
November 21, 2012
January 8, 2016
 
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 (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.
Expanded Access
Drug: Ruxolitinib
Ruxolitinib 10 mg tablets twice daily
Other Name: Jakafi
Not Provided
 
Available
Contact: Karyn Gordon, BSN 314-362-0156 kgordon@dom.wustl.edu
Contact: Jennifer Riggs 314-362-9903 jriggs@dom.wustl.edu
United States
 
 
NCT01730755
Washington University School of Medicine
Washington University School of Medicine
Incyte Corporation
Not Provided
Washington University School of Medicine
January 2016