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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01243944
Registration number
NCT01243944
Ethics application status
Date submitted
17/11/2010
Date registered
19/11/2010
Date last updated
6/03/2019
Titles & IDs
Public title
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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Scientific title
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)
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Secondary ID [1]
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CINC424B2301
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Polycythemia Vera
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Condition category
Condition code
Blood
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Haematological diseases
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Blood
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0
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Other blood disorders
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Intervention/exposure
Study type
Interventional(has expanded access)
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Description of intervention(s) / exposure
Treatment: Drugs - ruxolitinib tablets
Other interventions - Best Available Therapy (BAT)
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
Other: 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.
Treatment: Drugs: 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 interventions: 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.
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Intervention code [1]
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Treatment: Drugs
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Intervention code [2]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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The Percentage of Participants Achieving a Primary Response at Week 32
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Assessment method [1]
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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).
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Timepoint [1]
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32 Weeks
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Secondary outcome [1]
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The Percentage of Participants Achieving a Durable Primary Response at Week 48
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Assessment method [1]
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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.
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Timepoint [1]
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48 Weeks
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Secondary outcome [2]
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The Percentage of Participants Achieving Complete Hematological Remission at Week 32
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Assessment method [2]
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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.
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Timepoint [2]
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32 Weeks
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Secondary outcome [3]
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The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48
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Assessment method [3]
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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.
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Timepoint [3]
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48 Weeks
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Secondary outcome [4]
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The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48
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Assessment method [4]
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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.
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Timepoint [4]
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48 Weeks
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Secondary outcome [5]
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The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48
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Assessment method [5]
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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.
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Timepoint [5]
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48 Weeks
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Secondary outcome [6]
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Estimated Duration of the Primary Response
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Assessment method [6]
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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.
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Timepoint [6]
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Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study
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Secondary outcome [7]
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The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32
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Assessment method [7]
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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.
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Timepoint [7]
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32 Weeks
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Secondary outcome [8]
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The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48
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Assessment method [8]
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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.
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Timepoint [8]
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48 Weeks
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Secondary outcome [9]
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Estimated Duration of the Complete Hematological Remission
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Assessment method [9]
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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.
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Timepoint [9]
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Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study
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Secondary outcome [10]
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Duration of the Absence of Phlebotomy Eligibility
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Assessment method [10]
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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.
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Timepoint [10]
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256 Weeks
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Secondary outcome [11]
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Duration of Reduction in Spleen Volume
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Assessment method [11]
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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.
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Timepoint [11]
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256 Weeks
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Secondary outcome [12]
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Duration of The Overall Clinicohematologic Response
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Assessment method [12]
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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.
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Timepoint [12]
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256 Weeks
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Eligibility
Key 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
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
27/10/2010
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Date of last participant enrolment
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Actual
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Date of last data collection
Anticipated
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Actual
9/02/2018
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Sample size
Target
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Accrual to date
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Final
222
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Recruitment in Australia
Recruitment state(s)
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- Brisbane
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- Parkville
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- Tweed Heads
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- Brisbane
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- Parkville
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- Tweed Heads
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Incyte Corporation
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Address
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Other collaborator category [1]
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Commercial sector/Industry
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Name [1]
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Novartis Pharmaceuticals
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Ethics approval
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Summary
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).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01243944
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Srdan Verstovsek, MD,PhD
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Address
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M.D. Anderson Cancer Center
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01243944
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