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)

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Incyte Corporation
ClinicalTrials.gov Identifier:
NCT01243944
First received: November 17, 2010
Last updated: April 8, 2015
Last verified: April 2015
Results First Received: December 22, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Polycythemia Vera
Interventions: Drug: ruxolitinib tablets
Other: Best Available Therapy (BAT)

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

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

Reporting Groups
  Description
Ruxolitinib Starting dose of 10 mg 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 Best Available Therapy (BAT) will be selected by the Investigator for each subject. 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.

Participant Flow:   Overall Study
    Ruxolitinib     Best Available Therapy  
STARTED     110     111 [1]
COMPLETED     93 [2]   3 [2]
NOT COMPLETED     17     108  
Adverse Event                 4                 2  
Lack of Efficacy                 0                 98  
Disease progression                 5                 1  
Patient decision                 6                 5  
Physician Decision                 2                 2  
[1] One subject withdrew consent 5 days after randomization and was not treated while on study
[2] These are the numbers of subjects who completed 48 weeks of treatment.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
All enrolled participants, regardless treated or not.

Reporting Groups
  Description
Ruxolitinib 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 Best Available Therapy (BAT) will be selected by the Investigator for each subject. 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 Total of all reporting groups

Baseline Measures
    Ruxolitinib     Best Available Therapy     Total  
Number of Participants  
[units: participants]
  110     112     222  
Age  
[units: years]
Mean (Standard Deviation)
  61.1  (10.48)     59.1  (10.25)     60.1  (10.39)  
Age, Customized  
[units: participants]
     
< 60 years     49     54     103  
≥ 60 years     61     58     119  
Gender  
[units: participants]
     
Female     44     32     76  
Male     66     80     146  
Race/Ethnicity, Customized  
[units: participants]
     
White/Caucasian     98     96     194  
Black/African American     1     0     1  
Asian     11     16     27  
Weight  
[units: kg]
Mean (Standard Deviation)
  76.6  (14.09)     79.0  (17.34)     77.8  (15.83)  
Height [1]
[units: cm]
Mean (Standard Deviation)
  172.2  (8.44)     173.3  (9.79)     172.8  (9.14)  
Body Mass Index (BMI)  
[units: kg/m^2]
Mean (Standard Deviation)
  25.8  (3.82)     26.1  (4.24)     25.9  (4.03)  
ECOG performance status [2]
[units: participants]
     
0     76     77     153  
1     31     34     65  
2     3     1     4  
[1] Height was missing at study entry for one patient in the ruxolitinib arm.
[2] 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



  Outcome Measures
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1.  Primary:   The Percentage of Subjects Achieving a Primary Response at Week 32   [ Time Frame: 32 Weeks ]

2.  Secondary:   The Percentage of Subjects Achieving a Durable Primary Response at Week 48   [ Time Frame: 48 Weeks ]

3.  Secondary:   The Percentage of Subjects Achieving Complete Hematological Remission at Week 32   [ Time Frame: 32 Weeks ]

4.  Secondary:   The Percentage of Subjects Who Achieved a Durable Complete Hematological Remission at Week 48   [ Time Frame: 48 Weeks ]

5.  Secondary:   The Percentage of Subjects Who Achieved a Durable Hematocrit Control at Week 48   [ Time Frame: 48 Weeks ]

6.  Secondary:   The Percentage of Subjects Who Achieved Durable Spleen Volume Reduction at Week 48   [ Time Frame: 48 Weeks ]

7.  Secondary:   The Percentage of Subjects Who Achieved Overall Clinicohematologic Response at Week 32   [ Time Frame: 32 Weeks ]

8.  Secondary:   The Percentage of Subjects Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48   [ Time Frame: 48 Weeks ]

9.  Secondary:   Estimated Duration of the Primary Response   [ Time Frame: Through study completion ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

10.  Secondary:   Estimated Duration of the Overall Clinicohematologic Response   [ Time Frame: Through study completion ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


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


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


Results Point of Contact:  
Name/Title: Study Director
Organization: Incyte Corporation
phone: 855-463-3463
e-mail: medinfo@incyte.com


No publications provided by Incyte Corporation

Publications automatically indexed to this study:

Responsible Party: Incyte Corporation
ClinicalTrials.gov Identifier: NCT01243944     History of Changes
Other Study ID Numbers: CINC424B2301
Study First Received: November 17, 2010
Results First Received: December 22, 2014
Last Updated: April 8, 2015
Health Authority: United States: Food and Drug Administration