Overview

This trial is active, not recruiting.

Conditions polycythemia vera, essential thrombocythemia
Treatment ruxolitinib
Phase phase 2
Target JAK
Sponsor Incyte Corporation
Start date July 2008
End date June 2010
Trial size 73 participants
Trial identifier NCT00726232, INCB 18424-256

Summary

To evaluate the safety and efficacy profile of different treatment regimens of Ruxolitinib (INCB018424) administered to two groups of patients; those with polycythemia vera (PV) and those with essential thrombocythemia (ET). Patients in each group will be refractory to hydroxyurea or for whom hydroxyurea is contraindicated.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Participants received 10 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
ruxolitinib INCB018424
Ruxolitinib was administered orally and supplied as 5 mg and 25 mg tablets.
(Experimental)
Participants received 25 mg Ruxolitinib orally twice a day (BID) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
ruxolitinib INCB018424
Ruxolitinib was administered orally and supplied as 5 mg and 25 mg tablets.
(Experimental)
Participants received 50 mg Ruxolitinib orally once a day (QD) for 56 days (two 28-day cycles) during the dose-ranging phase. After patients completed 2 cycles of treatment at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis to achieve an optimal balance of efficacy and safety. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
ruxolitinib INCB018424
Ruxolitinib was administered orally and supplied as 5 mg and 25 mg tablets.

Primary Outcomes

Measure
Percentage of Polycythemia Vera Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR)
time frame: Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3
Percentage of Essential Thrombocythemia (ET) Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR)
time frame: Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3.

Secondary Outcomes

Measure
Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 12 Weeks
time frame: Baseline and Week 12 (Cycle 4, Day 1)
Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 24 Weeks
time frame: Baseline and Week 24 (Cycle 7, Day 1)
Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 36 Weeks
time frame: Baseline and Week 36 (Cycle 10, Day 1)
Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 4 Weeks
time frame: Baseline and 4 weeks (Cycle 2, Day 1)
Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 24 Weeks
time frame: Baseline and 24 weeks (Cycle 7, Day 1)
Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 36 Weeks
time frame: Baseline and 36 weeks (Cycle 10, Day 1)
Change From Baseline to Week 4 in Polycythemia Vera Symptoms
time frame: Baseline and Week 4 (Cycle 2, Day 1)
Change From Baseline to Week 4 in Essential Thrombocythemia Symptoms
time frame: Baseline and Week 4 (Cycle 2, Day 1)
Change From Baseline to Week 4 in Health-related Quality of Life
time frame: Baseline and Week 4 (Cycle 2, Day 1)

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Confirmed diagnosis of polycythemia vera or essential thrombocythemia as determined by treating physician - Disease refractory to hydroxyurea or for whom treatment with hydroxyurea is contraindicated or have refused further treatment with hydroxyurea due to side effects. - Patient meets baseline clinical lab parameters Exclusion Criteria: - Treatment with interferon alpha or anagrelide within 7 days and hydroxyurea within 1 day of starting INCB018424. - Patients diagnosed with another malignancy unless the malignancy was cervical intraepithelial neoplasia or basal or squamous cell skin cancer and the patient has been disease free for > 3 years - Patients receiving therapy with intermediate or high dose steroids greater than the equivalent of 10 mg prednisone per day - Clinically significant cardiac disease (New York Heart Association (NYHA) Class III or IV)

Additional Information

Official title An Open-Label Study to Determine the Safety and Efficacy of INCB018424 in Patients With Advanced Polycythemia Vera or Essential Thrombocythemia Refractory to Hydroxyurea
Description The study consisted of a 2-stage design, which included a dose-ranging phase (during which patients received treatment at their randomized dose) and an expansion phase (after adjustment of dose/regimen to achieve an optimal balance of efficacy and safety). During the dose-ranging phase, patients in each disease group (PV or ET) were randomly assigned in a 1:1:1 ratio independent of each other to receive 1 of 3 treatment regimens with Ruxolitinib, 10 mg twice daily (bid), 25 mg bid, or 50 mg once daily (qd). After patients completed 2 cycles of treatment with Ruxolitinib at the randomized dose, Investigators were permitted to adjust the dose/regimen on an individual basis using their discretion in order to achieve an optimal balance of efficacy and safety. During the expansion phase (ie, after optimization of dose), additional patients with PV or ET were enrolled to receive Ruxolitinib at the dose that was selected upon review of data from the dose-ranging phase. Treatment continued until a patient met a withdrawal criterion, had intolerable toxicity, progression of disease, or withdrew consent.
Trial information was received from ClinicalTrials.gov and was last updated in April 2016.
Information provided to ClinicalTrials.gov by Incyte Corporation.