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Trial record 20 of 22 for:    "Bone Marrow Cancer" | "Protein Kinase Inhibitors"

Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 (INCB018424) Tablets Versus Best Available Care: (The RESPONSE Trial)

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.
 
ClinicalTrials.gov Identifier: NCT01243944
Recruitment Status : Completed
First Posted : November 19, 2010
Results First Posted : March 6, 2015
Last Update Posted : March 6, 2019
Sponsor:
Collaborator:
Novartis Pharmaceuticals
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 Polycythemia Vera
Interventions Drug: ruxolitinib tablets
Other: Best Available Therapy (BAT)
Enrollment 222
Recruitment Details Participants may be treated beyond 256 weeks due to the 14 day visit window.
Pre-assignment Details  
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description Starting dose of 10 mg twice a day (BID) with individualized dose titration ranging from 5 mg once a day (QD) to 25 mg BID based on safety and efficacy Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Period Title: Overall Study
Started 110 112 [1]
Treated 110 111
Followed for Survival 22 21
Completed 65 61 [2]
Not Completed 45 51
Reason Not Completed
Adverse Event             15             13
Lost to Follow-up             2             1
Physician Decision             2             7
Disease progression             12             11
Protocol deviation             1             1
Participant decision             10             15
Death             2             3
Non-compliance with study treatment             1             0
[1]
One participant withdrew consent 5 days after randomization to BAT & wasn't treated while on study.
[2]
Study evaluation completion is reported for participants that were treated & those never treatment.
Arm/Group Title Ruxolitinib Best Available Therapy Total
Hide Arm/Group Description Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. Total of all reporting groups
Overall Number of Baseline Participants 110 112 222
Hide Baseline Analysis Population Description
All enrolled participants, regardless treated or not.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 110 participants 112 participants 222 participants
61.1  (10.48) 59.1  (10.25) 60.1  (10.39)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 110 participants 112 participants 222 participants
< 60 years 49 54 103
≥ 60 years 61 58 119
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 110 participants 112 participants 222 participants
Female
44
  40.0%
32
  28.6%
76
  34.2%
Male
66
  60.0%
80
  71.4%
146
  65.8%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 110 participants 112 participants 222 participants
White/Caucasian 98 96 194
Black/African American 1 0 1
Asian 11 16 27
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 110 participants 112 participants 222 participants
76.6  (14.09) 79.0  (17.34) 77.8  (15.83)
Height   [1] 
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 110 participants 112 participants 222 participants
172.2  (8.44) 173.3  (9.79) 172.8  (9.14)
[1]
Measure Description: Height was missing at study entry for one participant in the ruxolitinib arm.
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 110 participants 112 participants 222 participants
25.8  (3.82) 26.1  (4.24) 25.9  (4.03)
ECOG performance status   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 110 participants 112 participants 222 participants
0 76 77 153
1 31 34 65
2 3 1 4
[1]
Measure Description: Eastern Cooperative Oncology Group (ECOG) - ECOG Status: 0=Fully active, able to carry on all pre-disease performance without restriction; 1=Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature; 2=Ambulatory and capable of all selfcare but unable to carry out any work activities up and about more than 50% of waking hours
1.Primary Outcome
Title The Percentage of Participants Achieving a Primary Response at Week 32
Hide Description Primary response was defined as having achieved hematocrit control (the absence of phlebotomy eligibility beginning at the Week 8 visit and continuing through Week 32) and Spleen Volume Reduction (a greater than or equal to 35% reduction from baseline in spleen volume at Week 32).
Time Frame 32 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
22.7
(15.3 to 31.7)
0.9
(0.0 to 4.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ruxolitinib, Best Available Therapy
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value < 0.0001
Comments [Not Specified]
Method Exact Cochran-Mantel-Haenszel
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio, log
Estimated Value 32.67
Confidence Interval (2-Sided) 95%
5.04 to 1337
Estimation Comments [Not Specified]
2.Secondary Outcome
Title The Percentage of Participants Achieving a Durable Primary Response at Week 48
Hide Description Durable Primary Response was defined as any participant who achieved the primary outcome measure and who maintained their response up to 48 weeks after randomization.
Time Frame 48 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
20.0
(13.0 to 28.7)
0.9
(0.0 to 4.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ruxolitinib, Best Available Therapy
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Exact Cochran-Mantel-Haenszel
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 28.01
Confidence Interval (2-Sided) 95%
4.24 to 1144
Estimation Comments [Not Specified]
3.Secondary Outcome
Title The Percentage of Participants Achieving Complete Hematological Remission at Week 32
Hide Description Complete Hematological Remission at Week 32 was defined as any participant who achieved hematocrit control with a platelet count less than or equal to 400 X 10^9/L and a white blood cell count less than or equal to 10 X 10^9/L.
Time Frame 32 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
23.6
(16.1 to 32.7)
8.0
(3.7 to 14.7)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Ruxolitinib, Best Available Therapy
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0016
Comments [Not Specified]
Method Exact Cochran-Mantel-Haenszel
Comments P-value was calculated using stratified exact Cochran-Mantel-Haenszel test by adjusting for the WBC/platelet status (abnormal vs normal) at baseline.
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 3.57
Confidence Interval (2-Sided) 95%
1.50 to 9.06
Estimation Comments [Not Specified]
4.Secondary Outcome
Title The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48
Hide Description Durable Complete Hematological Remission was defined as any participant who achieved Complete Hematological Remission at Week 32 and maintained their response up to 48 weeks after randomization.
Time Frame 48 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
20.9
(13.7 to 29.7)
0.9
(0.0 to 4.9)
5.Secondary Outcome
Title The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48
Hide Description Durable Hematocrit Control was defined as any participant who achieved phlebotomy eligibility independence from Week 8 to Week 32 and maintained hematocrit control up to 48 weeks after randomization.
Time Frame 48 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
54.5
(44.8 to 64.1)
1.8
(0.2 to 6.3)
6.Secondary Outcome
Title The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48
Hide Description Durable Spleen Volume Reduction was defined as a participant who achieved at least 35% reduction from baseline in spleen volume at Week 32 and maintained that response 48 weeks after randomization.
Time Frame 48 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
37.3
(28.2 to 47.0)
0.9
(0.0 to 4.9)
7.Secondary Outcome
Title Estimated Duration of the Primary Response
Hide Description

Duration of the primary response is defined as the time from the first occurrence when both components of the primary endpoint are met until the date of the first documented disease progression (end of response).

Kaplan-Meier estimates are provided for duration of primary response.

Time Frame Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of response was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Overall Number of Participants Analyzed 110
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: probability
16 weeks
1.00 [1] 
(NA to NA)
32 weeks
1.00 [1] 
(NA to NA)
48 weeks
0.92
(0.72 to 0.98)
64 weeks
0.92
(0.72 to 0.98)
80 weeks
0.92
(0.72 to 0.98)
96 weeks
0.88
(0.67 to 0.96)
112 weeks
0.84
(0.62 to 0.94)
128 weeks
0.84
(0.62 to 0.94)
144 weeks
0.84
(0.62 to 0.94)
160 weeks
0.79
(0.57 to 0.91)
176 weeks
0.79
(0.57 to 0.91)
192 weeks
0.74
(0.51 to 0.88)
208 weeks
0.74
(0.51 to 0.88)
224 weeks
0.74
(0.51 to 0.88)
240 weeks
NA [2] 
(NA to NA)
256 weeks
NA [3] 
(NA to NA)
[1]
A confidence interval cannot be estimated at this time-point because no one had lost response.
[2]
No additional progression was experienced within population at week 240.
[3]
No additional progression was experienced within population at week 256.
8.Secondary Outcome
Title The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32
Hide Description Overall Clinicohematologic Response is defined as any participant who achieved a complete or partial clinicohematologic response per the European LeukemiaNet modified criteria for response in polycythemia vera (PV). A Complete Response (CR) is defined as: hematocrit control, spleen volume reduction at least 35% from baseline, platelet count less than or equal to 400 x 10(9)/L, and white blood cell count less than or equal to 10 x 10(9)/L. A Partial Response (PR) is defined as hematocrit control or response in all 3 of the other criteria.
Time Frame 32 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Unit of Measure: percentage of participants
Complete response rate 8.2 0.9
Partial response rate 54.5 18.8
9.Secondary Outcome
Title The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48
Hide Description Durable Complete or Partial Clinicohematologic Response was defined as any participant who achieved complete or partial clinicohematologic response per the European LeukemiaNet modified criteria for response in polycythemia vera at Week 32 and maintained that response 48 weeks after randomization.
Time Frame 48 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization.
Arm/Group Title Ruxolitinib Best Available Therapy
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
Overall Number of Participants Analyzed 110 112
Measure Type: Number
Unit of Measure: percentage of participants
Complete response rate 7.3 0.9
Partial response rate 50.9 0.9
10.Secondary Outcome
Title Estimated Duration of the Complete Hematological Remission
Hide Description

Duration of the complete hematological remission is defined as the time from the first occurrence of complete hematological remission until the date of the first documented progression (end of response).

Kaplan-Meier estimates are provided for duration of complete hematological remission.

Time Frame Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of response was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Overall Number of Participants Analyzed 110
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: probability
16 weeks
1.00 [1] 
(NA to NA)
32 weeks
1.00 [1] 
(NA to NA)
48 weeks
0.88
(0.66 to 0.96)
64 weeks
0.83
(0.61 to 0.93)
80 weeks
0.74
(0.51 to 0.87)
96 weeks
0.74
(0.51 to 0.87)
112 weeks
0.69
(0.46 to 0.84)
128 weeks
0.69
(0.46 to 0.84)
144 weeks
0.65
(0.41 to 0.81)
160 weeks
0.65
(0.41 to 0.81)
176 weeks
0.55
(0.32 to 0.73)
192 weeks
0.55
(0.32 to 0.73)
208 weeks
0.55
(0.32 to 0.73)
224 weeks
0.55
(0.32 to 0.73)
240 weeks
NA [2] 
(NA to NA)
256 weeks
NA [3] 
(NA to NA)
[1]
A confidence interval cannot be estimated at this time-point because no one had lost response.
[2]
No additional progression was experienced within population at week 240.
[3]
No additional progression was experienced within population at week 256.
11.Secondary Outcome
Title Duration of the Absence of Phlebotomy Eligibility
Hide Description Duration of the absence of phlebotomy eligibility is defined as the time from the first occurrence of absence of phlebotomy eligibility until the date of the first documented progression.
Time Frame 256 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of the absence of phlebotomy eligibility was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Overall Number of Participants Analyzed 110
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: probability
16 weeks
1.00 [1] 
(NA to NA)
32 weeks
1.00 [1] 
(NA to NA)
48 weeks
0.97
(0.88 to 0.99)
64 weeks
0.92
(0.82 to 0.97)
80 weeks
0.91
(0.80 to 0.96)
96 weeks
0.91
(0.80 to 0.96)
112 weeks
0.87
(0.76 to 0.93)
128 weeks
0.84
(0.72 to 0.91)
144 weeks
0.84
(0.72 to 0.91)
160 weeks
0.82
(0.70 to 0.90)
176 weeks
0.79
(0.66 to 0.87)
192 weeks
0.77
(0.64 to 0.86)
208 weeks
0.73
(0.60 to 0.83)
224 weeks
0.73
(0.60 to 0.83)
240 weeks
0.73
(0.60 to 0.83)
256 weeks
0.73
(0.60 to 0.83)
[1]
A confidence interval cannot be estimated at this time-point because no one had lost response.
12.Secondary Outcome
Title Duration of Reduction in Spleen Volume
Hide Description Duration of spleen volume reduction is defined as the time from the first occurrence of a >=35% reduction from baseline in spleen volume until the date of the first documented progression.
Time Frame 256 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of the reduction in spleen volume was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Overall Number of Participants Analyzed 110
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: probability
16 weeks
1.00 [1] 
(NA to NA)
32 weeks
1.00 [1] 
(NA to NA)
48 weeks
1.00 [1] 
(NA to NA)
64 weeks
1.00 [1] 
(NA to NA)
80 weeks
1.00 [1] 
(NA to NA)
96 weeks
0.98
(0.84 to 1.00)
112 weeks
0.95
(0.82 to 0.99)
128 weeks
0.95
(0.82 to 0.99)
144 weeks
0.95
(0.82 to 0.99)
160 weeks
0.93
(0.79 to 0.98)
176 weeks
0.93
(0.79 to 0.98)
192 weeks
0.93
(0.79 to 0.98)
208 weeks
0.87
(0.66 to 0.95)
224 weeks
0.72
(0.34 to 0.91)
240 weeks
NA [2] 
(NA to NA)
256 weeks
NA [3] 
(NA to NA)
[1]
A confidence interval cannot be estimated at this time-point because none of the participants had progressed.
[2]
No additional progression was experienced within population at week 240.
[3]
No additional progression was experienced within population at week 256.
13.Secondary Outcome
Title Duration of The Overall Clinicohematologic Response
Hide Description Duration of the overall clinicohematologic response was defined as the time from the first occurrence of complete response (CR) or partial response (PR) until the date of the first documented disease progression.
Time Frame 256 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) comprises all participants to whom study treatment had been assigned by randomization. Duration of the overall clinicohematologic response was pre-specified in the protocol to be analyzed for the ruxolitinib arm only considering the study design which allowed crossover for the BAT arm beginning at Week 32.
Arm/Group Title Ruxolitinib
Hide Arm/Group Description:
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
Overall Number of Participants Analyzed 110
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: probability
16 weeks
1.00 [1] 
(NA to NA)
32 weeks
0.99
(0.90 to 1.00)
48 weeks
0.96
(0.87 to 0.99)
64 weeks
0.91
(0.81 to 0.96)
80 weeks
0.88
(0.78 to 0.94)
96 weeks
0.88
(0.78 to 0.94)
112 weeks
0.85
(0.74 to 0.92)
128 weeks
0.82
(0.71 to 0.89)
144 weeks
0.82
(0.71 to 0.89)
160 weeks
0.80
(0.69 to 0.88)
176 weeks
0.75
(0.63 to 0.84)
192 weeks
0.70
(0.57 to 0.80)
208 weeks
0.67
(0.54 to 0.77)
224 weeks
0.67
(0.54 to 0.77)
240 weeks
0.67
(0.54 to 0.77)
256 weeks
0.67
(0.54 to 0.77)
[1]
A confidence interval cannot be estimated at this time-point because no one had lost response.
Time Frame The initial data reported was for the study up to Week 32. This time frame defines the comparative phase of the study where the majority of participants remained on their original randomized assignment and exposure between ruxolitinib and best available therapy was similar. Data inclusive of end of study was reported up to week 256 for Ruxolitinib group. Long term Follow Up for BAT was not reported as all patients have completed the study or crossed over to Ruxolitinib group by week 80.
Adverse Event Reporting Description

Safety population: All participants who were enrolled and took at least 1 dose of study medication.

The initial data reported at Week 32 was based on MedDRA 15.1; the remaining data reported for end of study was based on MedDRA 20.1.

 
Arm/Group Title Ruxolitinib - Through Week 32 Best Available Therapy - Through Week 32 Ruxolitinib - Week 256 Close Out
Hide Arm/Group Description Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements. Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
All-Cause Mortality
Ruxolitinib - Through Week 32 Best Available Therapy - Through Week 32 Ruxolitinib - Week 256 Close Out
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Ruxolitinib - Through Week 32 Best Available Therapy - Through Week 32 Ruxolitinib - Week 256 Close Out
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   15/110 (13.64%)   10/111 (9.01%)   44/110 (40.00%) 
Blood and lymphatic system disorders       
Leukocytosis  1  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Neutropenia  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Thrombocytopenia  1  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Cardiac disorders       
Atrial fibrillation  2  0/110 (0.00%)  1/111 (0.90%)  3/110 (2.73%) 
Cardiac failure congestive  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Acute myocardial infarction  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Tachycardia  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Atrioventricular block complete  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Eye disorders       
Cataract  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Glaucoma  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Retinal detachment  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Gastrointestinal disorders       
Dental necrosis  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Rectal haemorrhage  2  0/110 (0.00%)  0/111 (0.00%)  2/110 (1.82%) 
Anal fistula  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Gastric varices haemorrhage  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Haematochezia  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Ileus  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Incarcerated inguinal hernia  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Large intestinal stenosis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Varices oesophageal  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Volvulus  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
General disorders       
Chest pain  2  1/110 (0.91%)  0/111 (0.00%)  2/110 (1.82%) 
Hepatobiliary disorders       
Cholecystitis acute  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Cholelithiasis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Infections and infestations       
Bronchitis viral  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Diverticulitis  2  1/110 (0.91%)  1/111 (0.90%)  1/110 (0.91%) 
Pneumonia  2  1/110 (0.91%)  1/111 (0.90%)  5/110 (4.55%) 
Vulvovaginitis trichomonal  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Cellulitis  2  0/110 (0.00%)  1/111 (0.90%)  1/110 (0.91%) 
Gastroenteritis  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Appendicitis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Herpes zoster  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Lung infection  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Osteomyelitis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Peritonitis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Sepsis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Staphylococcal infection  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Tooth abscess  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Urinary tract infection  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Injury, poisoning and procedural complications       
Lumbar vertebral fracture  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Post procedural haemorrhage  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Splenic rupture  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Fall  2  0/110 (0.00%)  1/111 (0.90%)  1/110 (0.91%) 
Subdural haematoma  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Post procedural haematoma  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Rib fracture  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Metabolism and nutrition disorders       
Gout  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Dehydration  2  0/110 (0.00%)  0/111 (0.00%)  2/110 (1.82%) 
Musculoskeletal and connective tissue disorders       
Muscular weakness  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Intervertebral disc protrusion  2  0/110 (0.00%)  0/111 (0.00%)  3/110 (2.73%) 
Lumbar spinal stenosis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Musculoskeletal pain  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Spondylolisthesis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Basal cell carcinoma  2  1/110 (0.91%)  0/111 (0.00%)  3/110 (2.73%) 
Acute leukaemia  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Breast cancer  2  1/110 (0.91%)  0/111 (0.00%)  0/110 (0.00%) 
Myelofibrosis  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Malignant melanoma  2  0/110 (0.00%)  1/111 (0.90%)  1/110 (0.91%) 
Squamous cell carcinoma  2  0/110 (0.00%)  0/111 (0.00%)  4/110 (3.64%) 
Metastatic squamous cell carcinoma  2  0/110 (0.00%)  0/111 (0.00%)  2/110 (1.82%) 
Squamous cell carcinoma of skin  2  0/110 (0.00%)  0/111 (0.00%)  2/110 (1.82%) 
Adenocarcinoma gastric  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Carcinoma in situ of skin  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Chronic myeloid leukaemia  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Hairy cell leukaemia  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Invasive ductal breast carcinoma  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Keratoacanthoma  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Mediastinum neoplasm  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Metastases to central nervous system  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Metastasis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Neoplasm malignant  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Prostate cancer  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Rectosigmoid cancer  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Nervous system disorders       
Neurological symptom  1  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Cerebral infarction  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Metabolic encephalopathy  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Psychiatric disorders       
Attention deficit/hyperactivity disorder  1  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Renal and urinary disorders       
Bladder disorder  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Acute kidney injury  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Respiratory, thoracic and mediastinal disorders       
Chronic obstructive pulmonary disease  2  1/110 (0.91%)  0/111 (0.00%)  1/110 (0.91%) 
Pulmonary embolism  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Atelectasis  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Respiratory failure  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Vascular disorders       
Deep vein thrombosis  2  0/110 (0.00%)  1/111 (0.90%)  0/110 (0.00%) 
Hypotension  2  0/110 (0.00%)  0/111 (0.00%)  1/110 (0.91%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.1
2
Term from vocabulary, MedDRA 20.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Ruxolitinib - Through Week 32 Best Available Therapy - Through Week 32 Ruxolitinib - Week 256 Close Out
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   105/110 (95.45%)   104/111 (93.69%)   107/110 (97.27%) 
Blood and lymphatic system disorders       
Anaemia  1  20/110 (18.18%)  3/111 (2.70%)  38/110 (34.55%) 
Thrombocytopenia  1  9/110 (8.18%)  12/111 (10.81%)  19/110 (17.27%) 
Ear and labyrinth disorders       
Tinnitus  1  6/110 (5.45%)  3/111 (2.70%)  9/110 (8.18%) 
Vertigo  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Gastrointestinal disorders       
Diarrhoea  1  16/110 (14.55%)  8/111 (7.21%)  30/110 (27.27%) 
Abdominal pain  1  10/110 (9.09%)  13/111 (11.71%)  16/110 (14.55%) 
Constipation  1  9/110 (8.18%)  3/111 (2.70%)  14/110 (12.73%) 
Nausea  1  7/110 (6.36%)  4/111 (3.60%)  15/110 (13.64%) 
Abdominal pain upper  1  6/110 (5.45%)  5/111 (4.50%)  12/110 (10.91%) 
Abdominal distension  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Dyspepsia  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Vomiting  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
General disorders       
Fatigue  1  16/110 (14.55%)  17/111 (15.32%)  22/110 (20.00%) 
Asthenia  1  8/110 (7.27%)  12/111 (10.81%)  16/110 (14.55%) 
Oedema peripheral  1  7/110 (6.36%)  7/111 (6.31%)  9/110 (8.18%) 
Oedema  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Pyrexia  1  0/110 (0.00%)  0/111 (0.00%)  17/110 (15.45%) 
Infections and infestations       
Nasopharyngitis  1  10/110 (9.09%)  9/111 (8.11%)  19/110 (17.27%) 
Herpes zoster  1  7/110 (6.36%)  0/111 (0.00%)  19/110 (17.27%) 
Bronchitis  1  0/110 (0.00%)  0/111 (0.00%)  14/110 (12.73%) 
Influenza  1  0/110 (0.00%)  0/111 (0.00%)  13/110 (11.82%) 
Pharyngitis  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Upper respiratory tract infection  2  0/110 (0.00%)  0/111 (0.00%)  10/110 (9.09%) 
Urinary tract infection  1  0/110 (0.00%)  0/111 (0.00%)  10/110 (9.09%) 
Injury, poisoning and procedural complications       
Contusion  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Investigations       
Weight increased  1  6/110 (5.45%)  1/111 (0.90%)  26/110 (23.64%) 
Gamma-glutamyltransferase increased  1  6/110 (5.45%)  3/111 (2.70%)  9/110 (8.18%) 
Alanine aminotransferase increased  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Aspartate aminotransferase increased  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Blood cholesterol increased  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Metabolism and nutrition disorders       
Decreased appetite  1  3/110 (2.73%)  6/111 (5.41%)  9/110 (8.18%) 
Musculoskeletal and connective tissue disorders       
Muscle spasms  1  13/110 (11.82%)  5/111 (4.50%)  22/110 (20.00%) 
Arthralgia  1  8/110 (7.27%)  7/111 (6.31%)  24/110 (21.82%) 
Back pain  1  6/110 (5.45%)  4/111 (3.60%)  17/110 (15.45%) 
Myalgia  1  5/110 (4.55%)  8/111 (7.21%)  7/110 (6.36%) 
Bone pain  1  3/110 (2.73%)  6/111 (5.41%)  4/110 (3.64%) 
Musculoskeletal pain  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Osteoarthritis  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Pain in extremity  1  0/110 (0.00%)  0/111 (0.00%)  10/110 (9.09%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Basal cell carcinoma  1  0/110 (0.00%)  0/111 (0.00%)  16/110 (14.55%) 
Myelofibrosis  1  0/110 (0.00%)  0/111 (0.00%)  8/110 (7.27%) 
Squamous cell carcinoma of skin  1  0/110 (0.00%)  0/111 (0.00%)  7/110 (6.36%) 
Nervous system disorders       
Headache  1  18/110 (16.36%)  21/111 (18.92%)  25/110 (22.73%) 
Dizziness  1  13/110 (11.82%)  11/111 (9.91%)  17/110 (15.45%) 
Paraesthesia  1  5/110 (4.55%)  7/111 (6.31%)  7/110 (6.36%) 
Hypoaesthesia  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Neuralgia  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Neuropathy peripheral  1  0/110 (0.00%)  0/111 (0.00%)  6/110 (5.45%) 
Psychiatric disorders       
Insomnia  1  5/110 (4.55%)  6/111 (5.41%)  11/110 (10.00%) 
Respiratory, thoracic and mediastinal disorders       
Dyspnoea  1  11/110 (10.00%)  2/111 (1.80%)  19/110 (17.27%) 
Cough  1  9/110 (8.18%)  6/111 (5.41%)  20/110 (18.18%) 
Epistaxis  1  7/110 (6.36%)  3/111 (2.70%)  9/110 (8.18%) 
Skin and subcutaneous tissue disorders       
Pruritus  1  15/110 (13.64%)  25/111 (22.52%)  30/110 (27.27%) 
Night sweats  1  6/110 (5.45%)  9/111 (8.11%)  13/110 (11.82%) 
Vascular disorders       
Haematoma  1  6/110 (5.45%)  3/111 (2.70%)  11/110 (10.00%) 
Hypertension  1  0/110 (0.00%)  0/111 (0.00%)  17/110 (15.45%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 20.1
2
Term from vocabulary, MedDRA 15.1
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
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Incyte Corporation
Phone: 855-463-3463
EMail: medinfo@incyte.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Layout table for additonal information
Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT01243944     History of Changes
Other Study ID Numbers: CINC424B2301
First Submitted: November 17, 2010
First Posted: November 19, 2010
Results First Submitted: December 22, 2014
Results First Posted: March 6, 2015
Last Update Posted: March 6, 2019