Phase 3 N=222 Randomized Treatment

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)

Polycythemia Vera

Bottom Line

View on ClinicalTrials.gov: NCT01243944 ↗

Enrolled (actual)

222

Serious AEs

20.8%

Results posted

Mar 2015

Primary outcome: Primary: The Percentage of Participants Achieving a Primary Response at Week 32 — 22.7; 0.9 percentage of participants — p=< 0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: ruxolitinib tablets (Drug); Best Available Therapy (BAT) (Other)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Incyte Corporation
Primary completion: Jan 2014

Outcome Measures

Outcome	Result	p-value
PRIMARY The Percentage of Participants Achieving a Primary Response at Week 32	22.7; 0.9	< 0.0001 sig
SECONDARY The Percentage of Participants Achieving a Durable Primary Response at Week 48	20.0; 0.9	<0.0001 sig
SECONDARY The Percentage of Participants Achieving Complete Hematological Remission at Week 32	23.6; 8.0	0.0016 sig
SECONDARY The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48	20.9; 0.9	—
SECONDARY The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48	54.5; 1.8	—
SECONDARY The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48	37.3; 0.9	—
SECONDARY Estimated Duration of the Primary Response	1.00; 1.00; 0.92; 0.92; 0.92; 0.88	—
SECONDARY The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32	8.2; 0.9; 54.5; 18.8	—
SECONDARY The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48	7.3; 0.9; 50.9; 0.9	—
SECONDARY Estimated Duration of the Complete Hematological Remission	1.00; 1.00; 0.88; 0.83; 0.74; 0.74	—
SECONDARY Duration of the Absence of Phlebotomy Eligibility	1.00; 1.00; 0.97; 0.92; 0.91; 0.91	—
SECONDARY Duration of Reduction in Spleen Volume	1.00; 1.00; 1.00; 1.00; 1.00; 0.98	—
SECONDARY Duration of The Overall Clinicohematologic Response	1.00; 0.99; 0.96; 0.91; 0.88; 0.88	—

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).

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01243944). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.