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Trial record 20 of 23 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)

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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

Tracking Information
First Submitted Date  ICMJE November 17, 2010
First Posted Date  ICMJE November 19, 2010
Results First Submitted Date  ICMJE December 22, 2014
Results First Posted Date  ICMJE March 6, 2015
Last Update Posted Date March 6, 2019
Actual Study Start Date  ICMJE October 27, 2010
Actual Primary Completion Date January 15, 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 8, 2019)
The Percentage of Participants Achieving a Primary Response at Week 32 [ Time Frame: 32 Weeks ]
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).
Original Primary Outcome Measures  ICMJE
 (submitted: November 18, 2010)
Proportion of subjects achieving a response at Week 32. 'Response' is defined as having achieved both: (1) the absence of protocol-defined phlebotomy eligibility and (2) a ≥ 35% reduction from baseline in spleen volume as determined by imaging [ Time Frame: 32 weeks ]
Change History Complete list of historical versions of study NCT01243944 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 8, 2019)
  • The Percentage of Participants Achieving a Durable Primary Response at Week 48 [ Time Frame: 48 Weeks ]
    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.
  • The Percentage of Participants Achieving Complete Hematological Remission at Week 32 [ Time Frame: 32 Weeks ]
    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.
  • The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48 [ Time Frame: 48 Weeks ]
    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.
  • The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48 [ Time Frame: 48 Weeks ]
    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.
  • The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48 [ Time Frame: 48 Weeks ]
    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.
  • Estimated Duration of the Primary Response [ Time Frame: Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study ]
    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.
  • The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32 [ Time Frame: 32 Weeks ]
    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.
  • The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48 [ Time Frame: 48 Weeks ]
    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.
  • Estimated Duration of the Complete Hematological Remission [ Time Frame: Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study ]
    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.
  • Duration of the Absence of Phlebotomy Eligibility [ Time Frame: 256 Weeks ]
    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.
  • Duration of Reduction in Spleen Volume [ Time Frame: 256 Weeks ]
    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.
  • Duration of The Overall Clinicohematologic Response [ Time Frame: 256 Weeks ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: November 18, 2010)
  • Proportion of subjects achieving complete hematologic remission at Week 32. [ Time Frame: 32 weeks ]
  • Proportion of all randomized subjects who both achieve the Week 32 primary response endpoint and maintain response for ≥ 48 weeks from the time that response was initially documented. [ Time Frame: At least 80 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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)
Official Title  ICMJE Randomized, Open Label, Multicenter Phase III Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 Tablets Versus Best Available Care (The RESPONSE Trial)
Brief Summary This pivotal phase III trial (CINC424B2301) is designed to compare the efficacy and safety of ruxolitinib (INC424) to Best Available Therapy (BAT) in participants with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Polycythemia Vera
Intervention  ICMJE
  • Drug: ruxolitinib tablets
    Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg QD to 25 mg BID based on safety and efficacy
  • Other: Best Available Therapy (BAT)
    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.
    Other Names:
    • BAT could include:
    • Hydroxyurea
    • IFN/PEG-IFN
    • Pipobroman
    • Anagrelide
    • Lenalidomide
    • Pomalidomide
    • Observation only
Study Arms  ICMJE
  • Experimental: ruxolitinib tablets
    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
    Intervention: Drug: ruxolitinib tablets
  • Best Available Therapy
    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.
    Intervention: Other: Best Available Therapy (BAT)
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 19, 2015)
222
Original Estimated Enrollment  ICMJE
 (submitted: November 18, 2010)
300
Actual Study Completion Date  ICMJE February 9, 2018
Actual Primary Completion Date January 15, 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Participants diagnosed with PV for at least 24 weeks prior to screening according to the 2008 World Health Organization criteria
  • Participants resistant to or intolerant of hydroxyurea
  • Participants with a phlebotomy requirement
  • Participants with splenomegaly (palpable or non-palpable) and a spleen volume, as measured by MRI (or CT in applicable participants ), of greater than or equal to 450 cubic centimeters
  • Participants with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2

Exclusion Criteria:

  • Women who are pregnant or nursing
  • Participants with inadequate liver or renal function
  • Participants with significant bacterial, fungal, parasitic, or viral infection requiring treatment
  • Participants with an active malignancy within the past 5 years, excluding specific skin cancers
  • Participants with known active hepatitis or HIV positivity
  • Participants who have previously received treatment with a JAK inhibitor
  • Participants being treated with any investigational agent
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Belgium,   Canada,   China,   France,   Germany,   Hungary,   Italy,   Japan,   Korea, Republic of,   Netherlands,   Russian Federation,   Spain,   Thailand,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01243944
Other Study ID Numbers  ICMJE CINC424B2301
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Incyte Corporation
Study Sponsor  ICMJE Incyte Corporation
Collaborators  ICMJE Novartis Pharmaceuticals
Investigators  ICMJE
Study Director: Srdan Verstovsek, MD,PhD M.D. Anderson Cancer Center
Study Director: Mark Jones, MD Incyte Corporation
PRS Account Incyte Corporation
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP