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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 : Completed
First Posted : July 31, 2008
Results First Posted : February 24, 2012
Last Update Posted : September 28, 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
Enrollment 73
Recruitment Details  
Pre-assignment Details  
Arm/Group Title 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
Hide Arm/Group Description 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. 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. 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. 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. 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. 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.
Period Title: Overall Study
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
Reason Not Completed
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.
Arm/Group Title 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
Hide Arm/Group Description 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. 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. 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. 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. 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. 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 of all reporting groups
Overall Number of Baseline Participants 19 8 7 8 22 9 73
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 19 participants 8 participants 7 participants 8 participants 22 participants 9 participants 73 participants
Polycythemia vera group 56.3  (10.98) 57.0  (11.26) 51.0  (20.49) NA [1]   (NA) NA [1]   (NA) NA [1]   (NA) 55.4  (13.20)
Essential thrombocythemia group NA [2]   (NA) NA [2]   (NA) NA [2]   (NA) 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  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants 8 participants 7 participants 8 participants 22 participants 9 participants 73 participants
>= 65 5 3 2 1 3 3 17
< 65 14 5 5 7 19 6 56
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 8 participants 7 participants 8 participants 22 participants 9 participants 73 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  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants 8 participants 7 participants 8 participants 22 participants 9 participants 73 participants
Hematocrit ≥ 45% 10 7 6 1 4 0 28
Hematocrit < 45% 9 1 1 7 18 9 45
Platelet count  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants 8 participants 7 participants 8 participants 22 participants 9 participants 73 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  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 19 participants 8 participants 7 participants 8 participants 22 participants 9 participants 73 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
1.Primary Outcome
Title Percentage of Polycythemia Vera Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR)
Hide Description

For a confirmed response all criteria must have been sustained for at least 2 months.

CR:

  • Hematocrit < 45% in men and < 42% in women
  • No phlebotomy for 1 month
  • No palpable splenomegaly
  • White blood cells < 10 x 10^9/L with normal differential and platelets < 400 x 10^9/L
  • No sustained leucopenia or thrombocytopenia (>2 weeks)
  • No systemic PV symptoms (pruritus, night sweats, bone pain, fever, weight loss)

PR:

  • Hematocrit < 45% in men and < 42% in women
  • 50% reduction in phlebotomy requirements from 6 months before treatment started
  • 50% reduction in palpable splenomegaly
Time Frame Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Polycythemia Vera intent to treat population, including all patients who took at least 1 dose of study drug.
Arm/Group Title PV: Ruxolitinib 10 mg BID PV: Ruxolitinib 25 mg BID PV: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 19 8 7
Measure Type: Number
Unit of Measure: percentage of participants
58 50 57
2.Primary Outcome
Title Percentage of Essential Thrombocythemia (ET) Participants With a Confirmed Clinical Partial Response (PR) or Complete Response (CR)
Hide Description

For a confirmed response all criteria must have been sustained for at least 2 months.

Complete Clinical Response:

  • Platelet count < 400 x 10^9/L
  • White blood cell count < 10 x 10^9/L with normal differential and Hematocrit ≤ upper limit of normal
  • Absence of sustained (> 2 weeks) anemia or leucopenia based on institutional normal ranges
  • Absence of systemic ET symptoms (pruritus, bone pain, weakness, night sweats, paresthesias)
  • Absence of palpable splenomegaly

Partial Clinical Response:

  • Platelet count < 400 x 10^9/L
  • 50% reduction in palpable splenomegaly
Time Frame Assessed after 2 cycles (56 days) of treatment on Day 1 of Cycle 3.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Essential thrombocythemia intent to treat population, including all patients who took at least 1 dose of study drug. One patient in the 50 mg QD group did not have a response assessment at Cycle 3, Day 1.
Arm/Group Title ET: Ruxolitinib 10 mg BID ET: Ruxolitinib 25 mg BID ET: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 22 8
Measure Type: Number
Unit of Measure: percentage of participants
13 0 0
3.Secondary Outcome
Title Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 12 Weeks
Hide Description

The individual components of clinical response included:

  • Hematocrit (Hct) < 45% without phlebotomy
  • Absence of palpable splenomegaly
  • 50% reduction in spleen size
  • Platelet count ≤ 400 x 10^9/L
  • White blood cell (WBC) count ≤ 10 x 10^9/L
Time Frame Baseline and Week 12 (Cycle 4, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Polycythemia Vera intent to treat population for whom data was available.
Arm/Group Title PV: Ruxolitinib 10 mg BID PV: Ruxolitinib 25 mg BID PV: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 19 8 7
Measure Type: Number
Unit of Measure: percentage of participants
Hematocrit <45% Without Phlebotomy 95 88 86
Absence of palpable splenomegaly 68 50 57
50% reduction in spleen size 74 63 86
Platelet count ≤ 400 x 10^9/L 58 50 57
WBC count ≤ 10 x 10^9/L 68 63 43
4.Secondary Outcome
Title Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 24 Weeks
Hide Description

The individual components of clinical response included:

  • Hematocrit (Hct) < 45% without phlebotomy
  • Absence of palpable splenomegaly
  • 50% reduction in spleen size
  • Platelet count ≤ 400 x 10^9/L
  • White blood cell (WBC) count ≤ 10 x 10^9/L
Time Frame Baseline and Week 24 (Cycle 7, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Polycythemia Vera intent to treat population for whom data was available. 'N' indicates the number of patients for whom data was available for each component.
Arm/Group Title PV: Ruxolitinib 10 mg BID PV: Ruxolitinib 25 mg BID PV: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 19 8 7
Measure Type: Number
Unit of Measure: percentage of participants
Hematocrit <45% Without Phlebotomy [N= 19, 8, 7] 100 88 100
Absence of palpable splenomegaly [N= 18, 7, 7] 61 43 71
50% reduction in spleen size [N= 18, 7, 7] 78 71 100
Platelet count ≤ 400 x 10^9/L [N= 19, 8, 7] 58 88 86
WBC count ≤ 10 x 10^9/L [N= 19, 8, 7] 74 25 86
5.Secondary Outcome
Title Percentage of Polycythemia Vera Participants Who Achieved Individual Components of Clinical Response at 36 Weeks
Hide Description

The individual components of clinical response included:

  • Hematocrit (Hct) < 45% without phlebotomy
  • Absence of palpable splenomegaly
  • 50% reduction in spleen size
  • Platelet count ≤ 400 x 10^9/L
  • White blood cell (WBC) count ≤ 10 x 10^9/L
Time Frame Baseline and Week 36 (Cycle 10, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Polycythemia Vera intent to treat population for whom data was available. 'N' indicates the number of patients for whom data was available for each component.
Arm/Group Title PV: Ruxolitinib 10 mg BID PV: Ruxolitinib 25 mg BID PV: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 18 8 7
Measure Type: Number
Unit of Measure: percentage of participants
Hematocrit <45% Without Phlebotomy [N= 18, 8, 7] 100 88 100
Absence of palpable splenomegaly [N= 17, 7, 7] 71 57 86
50% reduction in spleen size [N= 17, 7, 7] 76 71 100
Platelet count ≤ 400 x 10^9/L [N= 18, 8, 7] 67 75 86
WBC count ≤ 10 x 10^9/L [N= 18, 8, 7] 67 25 71
6.Secondary Outcome
Title Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 4 Weeks
Hide Description

The individual components of clinical response included:

  • Platelet count ≤ 400 x 10^9/L
  • White blood cell (WBC) count ≤ 10 x 10^9/L
  • 50% reduction in spleen size
  • Absence of palpable splenomegaly
Time Frame Baseline and 4 weeks (Cycle 2, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Essential thrombocythemia intent to treat population. 'N' indicates the number of patients for whom data was available for each component.
Arm/Group Title ET: Ruxolitinib 10 mg BID ET: Ruxolitinib 25 mg BID ET: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 22 9
Measure Type: Number
Unit of Measure: percentage of participants
Platelet count ≤ 400 x 10^9/L [N= 8, 22, 9] 25 41 33
WBC count ≤ 10 x 10^9/L [N= 8, 22, 9] 100 100 100
50% reduction in spleen size [N= 6, 19, 8] 83 95 100
Absence of palpable splenomegaly [N= 6, 19, 8] 67 89 100
7.Secondary Outcome
Title Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 24 Weeks
Hide Description

The individual components of clinical response included:

  • Platelet count ≤ 400 x 10^9/L
  • White blood cell (WBC) count ≤ 10 x 10^9/L
  • 50% reduction in spleen size
  • Absence of palpable splenomegaly
Time Frame Baseline and 24 weeks (Cycle 7, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Essential thrombocythemia intent to treat population. 'N' indicates the number of patients for whom data was available for each component.
Arm/Group Title ET: Ruxolitinib 10 mg BID ET: Ruxolitinib 25 mg BID ET: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 22 9
Measure Type: Number
Unit of Measure: percentage of participants
Platelet count ≤ 400 x 10^9/L [N= 7, 21, 8] 14 5 0
WBC count ≤ 10 x 10^9/L [N= 7, 21, 8] 100 86 100
50% reduction in spleen size [N= 6, 19, 7] 100 100 100
Absence of palpable splenomegaly [N= 6, 19, 7] 100 95 100
8.Secondary Outcome
Title Percentage of Essential Thrombocythemia Participants Who Achieved Individual Components of Clinical Response at 36 Weeks
Hide Description

The individual components of clinical response included:

  • Platelet count ≤ 400 x 10^9/L
  • White blood cell (WBC) count ≤ 10 x 10^9/L
  • 50% reduction in spleen size
  • Absence of palpable splenomegaly
Time Frame Baseline and 36 weeks (Cycle 10, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Essential thrombocythemia intent to treat population. 'N' indicates the number of patients for whom data was available for each component.
Arm/Group Title ET: Ruxolitinib 10 mg BID ET: Ruxolitinib 25 mg BID ET: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 22 9
Measure Type: Number
Unit of Measure: percentage of participants
Platelet count ≤ 400 x 10^9/L [N= 7, 19, 7] 14 11 14
WBC count ≤ 10 x 10^9/L [N= 7, 19, 7] 100 79 86
50% reduction in spleen size [N= 6, 16, 6] 100 100 100
Absence of palpable splenomegaly [N= 6, 16, 6] 100 94 100
9.Secondary Outcome
Title Change From Baseline to Week 4 in Polycythemia Vera Symptoms
Hide Description

Patients were asked to rate their symptoms on a scale of 0 (none) to 10 (worse possible) for the prior week giving the worst level of symptoms experienced during the preceding 7 days. A negative change from baseline score indicates improvement in symptoms.

For patients with Polycythemia Vera, queried symptoms included fever, itching/pruritus, bone pain and night sweats.

Time Frame Baseline and Week 4 (Cycle 2, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Polycythemia Vera intent to treat population who had symptom scores > 0 at baseline for whom data was available.
Arm/Group Title PV: Ruxolitinib 10 mg BID PV: Ruxolitinib 25 mg BID PV: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 19 8 7
Mean (Standard Deviation)
Unit of Measure: scores on a scale
Itching (pruritus) [N= 13, 8, 5] -4.2  (3.63) -4.6  (1.85) -2.8  (4.09)
Bone pain [N= 12, 2, 3] -2.0  (1.95) -2.5  (0.71) -4.3  (2.08)
Fever [N= 2, 0, 2] -2.0  (1.41) NA [1]   (NA) -2.0  (1.41)
Night sweats [N= 9, 6, 3] -1.9  (2.52) -2.8  (3.19) -3.3  (1.15)
[1]
No patients in this group had a baseline score > 0.
10.Secondary Outcome
Title Change From Baseline to Week 4 in Essential Thrombocythemia Symptoms
Hide Description

Patients were asked to rate their symptoms on a scale of 0 (none) to 10 (worse possible) for the prior week giving the worst level of symptoms experienced during the preceding 7 days. A negative change from baseline score indicates improvement in symptoms.

For patients with essential thrombocythemia, queried symptoms included itching/pruritus, bone pain, night sweats, paresthesias (tingling or numbness), and weakness.

Time Frame Baseline and Week 4 (Cycle 2, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Essential Thrombocythemia intent to treat population who had symptom scores > 0 at baseline and for whom data was available.
Arm/Group Title ET: Ruxolitinib 10 mg BID ET: Ruxolitinib 25 mg BID ET: Ruxolitinib 50 mg QD
Hide Arm/Group Description:
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.
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.
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.
Overall Number of Participants Analyzed 8 22 8
Mean (Standard Deviation)
Unit of Measure: participants
Itching (pruritus) [N= 3, 10, 0] -2.7  (2.89) -1.2  (2.74) NA [1]   (NA)
Night Sweats [N= 1, 8, 3] -5.0 [2]   (NA) -1.3  (2.49) -4.0  (4.36)
Weakness [N= 3, 14, 3] -1.0  (1.00) -0.2  (2.46) -1.7  (2.52)
Bone pain [N= 2, 13, 3] -3.5  (2.12) -0.4  (2.26) -2.3  (4.93)
Paresthesia [N= 3, 14, 6] -1.7  (2.08) -1.6  (1.50) -2.8  (2.93)
[1]
No patients in this group had a Baseline symptom score > 0
[2]
Only one patient available for this analysis
11.Secondary Outcome
Title Change From Baseline to Week 4 in Health-related Quality of Life
Hide Description Health-related Quality of Life was assessed using the Global Health Status/Quality of Life Scale of the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30). This scale ranges from 0 to 100, with higher scores indicating higher quality of life.
Time Frame Baseline and Week 4 (Cycle 2, Day 1)
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Intent to treat population.
Arm/Group Title 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
Hide Arm/Group Description:
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.
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.
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.
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.
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.
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.
Overall Number of Participants Analyzed 19 8 7 8 22 9
Mean (Standard Deviation)
Unit of Measure: units on a scale
10.9  (10.80) 6.3  (14.0) 14.6  (17.78) -2.1  (10.5) 3.0  (27.6) 11.2  (23.4)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title 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
Hide Arm/Group Description 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. 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. 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. 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. 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. 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.
All-Cause Mortality
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
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
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
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/19 (10.53%)   2/8 (25.00%)   1/7 (14.29%)   1/8 (12.50%)   1/22 (4.55%)   3/9 (33.33%) 
Blood and lymphatic system disorders             
Anaemia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Thrombocytopenia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Cardiac disorders             
Atrial flutter  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Cardiac failure congestive  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Gastrointestinal disorders             
Gastric varices haemorrhage  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Gastrointestinal haemorrhage  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Hepatobiliary disorders             
Cholecystitis  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  1/22 (4.55%)  0/9 (0.00%) 
Infections and infestations             
Pneumonia  1  0/19 (0.00%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Bronchitis  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Renal neoplasm  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Nervous system disorders             
Headache  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Renal and urinary disorders             
Renal failure  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
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
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   19/19 (100.00%)   8/8 (100.00%)   7/7 (100.00%)   8/8 (100.00%)   22/22 (100.00%)   8/9 (88.89%) 
Blood and lymphatic system disorders             
Anaemia  1  16/19 (84.21%)  6/8 (75.00%)  3/7 (42.86%)  5/8 (62.50%)  19/22 (86.36%)  6/9 (66.67%) 
Thrombocytopenia  1  6/19 (31.58%)  4/8 (50.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Leukopenia  1  4/19 (21.05%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  2/22 (9.09%)  0/9 (0.00%) 
Lymphadenitis  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Neutropenia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Cardiac disorders             
Palpitations  1  2/19 (10.53%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  4/22 (18.18%)  0/9 (0.00%) 
Arrhythmia supraventricular  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Atrial fibrillation  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Supraventricular tachycardia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Tachycardia  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Cardiac failure  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Ear and labyrinth disorders             
Ear pain  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Eye disorders             
Conjunctivitis  1  1/19 (5.26%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Conjunctival haemorrhage  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Eye pain  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Myodesopsia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Photophobia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Vision blurred  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Visual impairment  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  2/22 (9.09%)  0/9 (0.00%) 
Gastrointestinal disorders             
Diarrhoea  1  4/19 (21.05%)  2/8 (25.00%)  1/7 (14.29%)  1/8 (12.50%)  4/22 (18.18%)  3/9 (33.33%) 
Vomiting  1  3/19 (15.79%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  1/22 (4.55%)  3/9 (33.33%) 
Abdominal pain  1  2/19 (10.53%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  2/22 (9.09%)  0/9 (0.00%) 
Constipation  1  0/19 (0.00%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  4/22 (18.18%)  1/9 (11.11%) 
Abdominal pain upper  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  1/8 (12.50%)  2/22 (9.09%)  0/9 (0.00%) 
Aphthous stomatitis  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Dental discomfort  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Gastrointestinal disorder  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Gastrooesophageal reflux disease  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Nausea  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  1/8 (12.50%)  2/22 (9.09%)  2/9 (22.22%) 
Toothache  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Abdominal pain lower  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  1/9 (11.11%) 
Dyspepsia  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  2/22 (9.09%)  0/9 (0.00%) 
Diverticulum  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
General disorders             
Pyrexia  1  4/19 (21.05%)  1/8 (12.50%)  1/7 (14.29%)  2/8 (25.00%)  4/22 (18.18%)  1/9 (11.11%) 
Asthenia  1  2/19 (10.53%)  2/8 (25.00%)  0/7 (0.00%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Fatigue  1  1/19 (5.26%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  2/22 (9.09%)  2/9 (22.22%) 
Oedema peripheral  1  1/19 (5.26%)  1/8 (12.50%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Chest pain  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Mucosal inflammation  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Hepatobiliary disorders             
Hyperbilirubinaemia  1  0/19 (0.00%)  0/8 (0.00%)  2/7 (28.57%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Infections and infestations             
Herpes zoster  1  2/19 (10.53%)  1/8 (12.50%)  1/7 (14.29%)  1/8 (12.50%)  1/22 (4.55%)  1/9 (11.11%) 
Upper respiratory tract infection  1  1/19 (5.26%)  1/8 (12.50%)  2/7 (28.57%)  0/8 (0.00%)  2/22 (9.09%)  0/9 (0.00%) 
Influenza  1  0/19 (0.00%)  2/8 (25.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Cystitis  1  1/19 (5.26%)  1/8 (12.50%)  0/7 (0.00%)  1/8 (12.50%)  1/22 (4.55%)  1/9 (11.11%) 
Gastroenteritis  1  1/19 (5.26%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Oral herpes  1  1/19 (5.26%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Pharyngitis  1  2/19 (10.53%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Bronchitis  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  2/8 (25.00%)  1/22 (4.55%)  0/9 (0.00%) 
Ear infection  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Epstein-Barr virus infection  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Respiratory tract infection  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Rhinitis  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Sinusitis  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Tooth infection  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Vulvovaginal mycotic infection  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Tooth abscess  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Injury, poisoning and procedural complications             
Foot fracture  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Patella fracture  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Investigations             
Weight increased  1  2/19 (10.53%)  1/8 (12.50%)  2/7 (28.57%)  2/8 (25.00%)  5/22 (22.73%)  3/9 (33.33%) 
Blood creatinine increased  1  1/19 (5.26%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  2/9 (22.22%) 
Alanine aminotransferase increased  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Aspartate aminotransferase increased  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Gamma-glutamyltransferase increased  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Transaminases increased  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Blood creatine phosphokinase increased  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  1/22 (4.55%)  0/9 (0.00%) 
Haematocrit decreased  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Metabolism and nutrition disorders             
Hyperuricaemia  1  2/19 (10.53%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  3/22 (13.64%)  0/9 (0.00%) 
Hypertriglyceridaemia  1  1/19 (5.26%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Diabetes mellitus  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Hypoalbuminaemia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Increased appetite  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  1/22 (4.55%)  0/9 (0.00%) 
Hypophosphataemia  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Musculoskeletal and connective tissue disorders             
Back pain  1  3/19 (15.79%)  1/8 (12.50%)  1/7 (14.29%)  1/8 (12.50%)  1/22 (4.55%)  1/9 (11.11%) 
Arthralgia  1  2/19 (10.53%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Neck pain  1  1/19 (5.26%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Joint stiffness  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Muscle spasms  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  1/8 (12.50%)  1/22 (4.55%)  1/9 (11.11%) 
Musculoskeletal pain  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  1/22 (4.55%)  1/9 (11.11%) 
Myalgia  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Pain in extremity  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  4/22 (18.18%)  1/9 (11.11%) 
Bone pain  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Muscular weakness  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  0/9 (0.00%) 
Nervous system disorders             
Dizziness  1  2/19 (10.53%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  3/22 (13.64%)  0/9 (0.00%) 
Headache  1  2/19 (10.53%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  6/22 (27.27%)  2/9 (22.22%) 
Dysgeusia  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Neuropathy peripheral  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Perineurial cyst  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Peripheral sensory neuropathy  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Post herpetic neuralgia  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Restless legs syndrome  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Paraesthesia  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Peripheral sensorimotor neuropathy  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Psychiatric disorders             
Insomnia  1  1/19 (5.26%)  0/8 (0.00%)  2/7 (28.57%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  2/19 (10.53%)  1/8 (12.50%)  1/7 (14.29%)  0/8 (0.00%)  4/22 (18.18%)  1/9 (11.11%) 
Dyspnoea  1  2/19 (10.53%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Oropharyngeal pain  1  0/19 (0.00%)  1/8 (12.50%)  2/7 (28.57%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Dyspnoea exertional  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Rhinorrhoea  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Sleep apnoea syndrome  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Skin and subcutaneous tissue disorders             
Pruritus  1  2/19 (10.53%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  2/22 (9.09%)  0/9 (0.00%) 
Erythema nodosum  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Mucocutaneous rash  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Rash pruritic  1  0/19 (0.00%)  1/8 (12.50%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Skin lesion  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  1/22 (4.55%)  1/9 (11.11%) 
Urticaria  1  0/19 (0.00%)  0/8 (0.00%)  1/7 (14.29%)  0/8 (0.00%)  0/22 (0.00%)  0/9 (0.00%) 
Vascular disorders             
Hypertension  1  1/19 (5.26%)  0/8 (0.00%)  0/7 (0.00%)  2/8 (25.00%)  0/22 (0.00%)  0/9 (0.00%) 
Hot flush  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  1/8 (12.50%)  0/22 (0.00%)  1/9 (11.11%) 
Hypertensive crisis  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Venous thrombosis limb  1  0/19 (0.00%)  0/8 (0.00%)  0/7 (0.00%)  0/8 (0.00%)  0/22 (0.00%)  1/9 (11.11%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
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.
Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study; provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
Results Point of Contact
Name/Title: Albert Assad, MD
Organization: Incyte Corporation
Phone: 1-855-463-3463
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: September 28, 2018