Ruxolitinib for Chuvash Polycythemia

NCT ID: NCT01730755

Last Updated: 2018-05-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NO_LONGER_AVAILABLE

Study Classification

EXPANDED_ACCESS

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Erythrocytosis, Familial, 2

Interventions

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Ruxolitinib

Ruxolitinib 10 mg tablets twice daily

Intervention Type DRUG

Other Intervention Names

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Jakafi

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of Chuvash polycythemia

Exclusion Criteria

* Unable to comprehend or unwilling to sign an informed consent form.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Incyte Corporation

INDUSTRY

Sponsor Role collaborator

Washington University School of Medicine

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Countries

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United States

Other Identifiers

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I-RUX-12-03

Identifier Type: -

Identifier Source: org_study_id

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