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)

Incyte

Status and phase: Completed; Phase 3

Conditions: Polycythemia Vera

Treatments: Drug: ruxolitinib tablets; Other: Best Available Therapy (BAT)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01243944

CINC424B2301

Details and patient eligibility

About

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).

Enrollment: 222 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

---

222 participants in 2 patient groups

ruxolitinib tablets

Experimental group

Description:

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

Treatment:

Drug: ruxolitinib tablets

Best Available Therapy

Other group

Description:

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:

Other: Best Available Therapy (BAT)