Open Label Ruxolitinib (INCB018424) in Patients With Myelofibrosis and Post Polycythemia Vera/Essential Thrombocythemia Myelofibrosis

Incyte

Status and phase: Completed; Phase 2; Phase 1

Conditions: Myelofibrosis; Polycythemia Vera; Thrombocytosis

Treatments: Drug: Ruxolitinib

Study type: Interventional

Funder types: Industry

Identifiers

NCT00509899

INCB 18424-251

Details and patient eligibility

About

To determine the safety, tolerability and effectiveness of ruxolitinib (INCB018424), administered orally to patients with Primary Myelofibrosis (PMF), Post Polycythemia Vera Myelofibrosis (PPV-MF) and Essential Thrombocythemia Myelofibrosis (PET-MF).

Full description

This is a multicenter, open-label, non-randomized, dose escalation study of ruxolitinib, a small molecule Janus kinase (JAK) inhibitor, administered orally to patients with PMF, PPEV-MF or PET-MF. The study is comprised of 3 parts:

Part 1: Dose escalation and determination of maximum tolerated dose (complete).

Part 2: Exploration of alternative dosing schedules (complete).

Part 3: Further evaluation of selected dose regimens, including additional response measures to explore effect of ruxolitinib on symptoms and other parameters including daily physical activity and long-term survival (ongoing).

Enrollment: 154 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosed with PMF or Post-PV/ET MF
• Patients with myelofibrosis requiring therapy
• Adequate bone marrow reserve

Exclusion criteria

• Received anti-cancer medications or investigational therapy in the past 14 days

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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154 participants in 1 patient group

Ruxolitinib

Experimental group

Description:

All participants received oral ruxolitinib. Patients began treatment with either 10 mg twice a day (bid), 15 mg bid, 25 mg bid, 50 mg bid, 25 mg once a day (qd), 50 mg qd, 100 mg qd, or 200 mg qd, depending on the time period when they entered the study. The doses were titrated based on efficacy and safety to a maximum of 25 mg bid for patients who entered the study after sufficient dosing information had been obtained to define the maximum dose for patients in the study. Patients could continue receiving treatment indefinitely if receiving benefit at a dose that continues to maintain benefit but does not exceed a maximum dose of 25 mg BID.

Treatment:

Drug: Ruxolitinib