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

November 7, 2012
November 21, 2012
May 14, 2018
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

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
No longer available
Contact information is only displayed when the study is recruiting subjects
United States
Washington University School of Medicine
Washington University School of Medicine
Incyte Corporation
Not Provided
Washington University School of Medicine
May 2018