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Study to Determine the Safety and Efficacy of Ruxolitinib (INCB018424) in Patients With Polycythemia Vera or Essential Thrombocythemia

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ClinicalTrials.gov Identifier: NCT00726232
Recruitment Status : Active, not recruiting
First Posted : July 31, 2008
Results First Posted : February 24, 2012
Last Update Posted : January 2, 2018
Sponsor:
Information provided by (Responsible Party):
Incyte Corporation

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: MPN (Myeloproliferative Neoplasms)
Intervention: Drug: Ruxolitinib

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
PV: Ruxolitinib 10 mg BID Participants with Polycythemia Vera 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.
PV: Ruxolitinib 25 mg BID Participants with Polycythemia Vera 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.
PV: Ruxolitinib 50 mg QD Participants with Polycythemia Vera 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.
ET: Ruxolitinib 10 mg BID Participants with Essential Thrombocythemia 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.
ET: Ruxolitinib 25 mg BID Participants with Essential Thrombocythemia 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.
ET: Ruxolitinib 50 mg QD Participants with Essential Thrombocythemia 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.

Participant Flow:   Overall Study
    PV: Ruxolitinib 10 mg BID   PV: Ruxolitinib 25 mg BID   PV: Ruxolitinib 50 mg QD   ET: Ruxolitinib 10 mg BID   ET: Ruxolitinib 25 mg BID   ET: Ruxolitinib 50 mg QD
STARTED   19 [1]   8   7   8   22 [2]   9 
COMPLETED   14 [3]   7   7   7   17   5 
NOT COMPLETED   5   1   0   1   5   4 
Withdrawal by Subject                2                0                0                0                0                2 
Adverse Event                1                1                0                1                2                1 
Disease progression                1                0                0                0                0                0 
No response                1                0                0                0                3                1 
[1] The starting dose selected for the PV expansion cohort was 10 mg bid hence the sample size is larger
[2] The starting dose selected for the ET expansion cohort was 25 mg bid hence the sample size is larger
[3] Represents patients ongoing in the study as of the data cut-off date of 18 June 2010.



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
PV: Ruxolitinib 10 mg BID Participants with Polycythemia Vera 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.
PV: Ruxolitinib 25 mg BID Participants with Polycythemia Vera 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.
PV: Ruxolitinib 50 mg QD Participants with Polycythemia Vera 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.
ET: Ruxolitinib 10 mg BID Participants with Essential Thrombocythemia 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.
ET: Ruxolitinib 25 mg BID Participants with Essential Thrombocythemia 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.
ET: Ruxolitinib 50 mg QD Participants with Essential Thrombocythemia 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.
Total Total of all reporting groups

Baseline Measures
   PV: Ruxolitinib 10 mg BID   PV: Ruxolitinib 25 mg BID   PV: Ruxolitinib 50 mg QD   ET: Ruxolitinib 10 mg BID   ET: Ruxolitinib 25 mg BID   ET: Ruxolitinib 50 mg QD   Total 
Overall Participants Analyzed 
[Units: Participants]
 19   8   7   8   22   9   73 
Age [1] [2] 
[Units: Years]
Mean (Standard Deviation)
             
Polycythemia vera group   56.3  (10.98)   57.0  (11.26)   51.0  (20.49)   NA [1]   NA [1]   NA [1]   55.4  (13.20) 
Essential thrombocythemia group   NA [2]   NA [2]   NA [2]   49.6  (17.89)   54.1  (11.55)   52.8  (13.48)   52.9  (13.19) 
[1] Age demographic data for the polycythemia vera group.
[2] Age demographic data for the essential thrombocythemia group.
Age, Customized 
[Units: Participants]
             
>= 65   5   3   2   1   3   3   17 
< 65   14   5   5   7   19   6   56 
Sex: Female, Male 
[Units: Participants]
Count of Participants
             
Female      11  57.9%      2  25.0%      4  57.1%      3  37.5%      13  59.1%      9 100.0%      42  57.5% 
Male      8  42.1%      6  75.0%      3  42.9%      5  62.5%      9  40.9%      0   0.0%      31  42.5% 
Hematocrit 
[Units: Participants]
             
Hematocrit ≥ 45%   10   7   6   1   4   0   28 
Hematocrit < 45%   9   1   1   7   18   9   45 
Platelet count 
[Units: Participants]
             
≥400 x 10^9 per liter   13   4   6   7   22   9   61 
< 400 x 10^9 per liter   6   4   1   1   0   0   12 
White blood cell count 
[Units: Participants]
             
≥ 10 x 10^9 per liter   12   7   6   2   7   2   36 
< 10 x 10^9 per liter   7   1   1   6   15   7   37 


  Outcome Measures

1.  Primary:   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 ]

2.  Primary:   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. ]

3.  Secondary:   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) ]

4.  Secondary:   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) ]

5.  Secondary:   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) ]

6.  Secondary:   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) ]

7.  Secondary:   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) ]

8.  Secondary:   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) ]

9.  Secondary:   Change From Baseline to Week 4 in Polycythemia Vera Symptoms   [ Time Frame: Baseline and Week 4 (Cycle 2, Day 1) ]

10.  Secondary:   Change From Baseline to Week 4 in Essential Thrombocythemia Symptoms   [ Time Frame: Baseline and Week 4 (Cycle 2, Day 1) ]

11.  Secondary:   Change From Baseline to Week 4 in Health-related Quality of Life   [ Time Frame: Baseline and Week 4 (Cycle 2, Day 1) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Albert Assad, MD
Organization: Incyte Corporation
phone: 1-855-463-3463
e-mail: aassad@incyte.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT00726232     History of Changes
Other Study ID Numbers: INCB 18424-256
First Submitted: July 29, 2008
First Posted: July 31, 2008
Results First Submitted: January 20, 2012
Results First Posted: February 24, 2012
Last Update Posted: January 2, 2018