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Study of the JAK Inhibitor Ruxolitinib Administered Orally to Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera-Myelofibrosis (PPV-MF) or Post-Essential Thrombocythemia-Myelofibrosis (PET-MF)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by Incyte Corporation
Information provided by (Responsible Party):
Incyte Corporation Identifier:
First received: March 14, 2011
Last updated: September 23, 2016
Last verified: September 2016
This is a Phase IB, open-label, dose-finding study of the JAK 1 and 2 inhibitor ruxolitinib in patients with myelofibrosis (MF). The study consists of two periods: the core study period, comprising the dose escalation stage and the safety extension phase up to Week 24, then the extension study period beyond Week 24 and up to 3 years, to further characterize the safety and efficacy of ruxolitinib in this patient population. The dose escalation phase will enroll successive cohorts of patients who receive increasing doses of ruxolitinib until the maximum safe starting dose (MSSD) is determined. In the safety expansion phase, additional patients will be treated with ruxolitinib at the MSSD defined during dose escalation. The primary objective is to establish the MSSD of ruxolitinib in patients with MF and starting platelet counts < 100 x 10 ^9/L

Condition Intervention Phase
Drug: Ruxolitinib
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Incyte Corporation:

Primary Outcome Measures:
  • To establish the MSSD of ruxolitinib in patients with MF and baseline platelet counts < 100,000 as measured through blood sampling. [ Time Frame: Measured at the completion of each consecutive dose cohort using a Bayesian logistic regression model. ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Characterize the safety of ruxolitinib by monitoring the frequency, duration and severity of adverse and serious adverse events. [ Time Frame: Baseline, weekly for the 1st month of the study and then every 2 weeks until study completion or early termination at week 156. In addition, safety will also be assessed through the spontaneous reporting of serious adverse events. ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 72
Study Start Date: March 2011
Estimated Study Completion Date: September 2020
Estimated Primary Completion Date: September 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Ruxolitinib Drug: Ruxolitinib

Starting dose of ruxolitinib for cohort 1 in dose escalation phase - 5mg twice a day (BID)

Doses will be increased a total of approximately 5mg for successive dosing cohorts based on baseline platelet count


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Require treatment for MF and classified at least as intermediate risk level 1 defined by the International Working Group.
  • Platelet count < 100x10 ^9/L at screening or at Study Day 1.

Exclusion Criteria:

  • Received platelet transfusion within 14 days prior to Screening evaluations.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01317875

Contact: Incyte Corporation Call Center 1.855.463.3463

  Show 24 Study Locations
Sponsors and Collaborators
Incyte Corporation
Study Director: Mark Jones, MD Incyte Corporation
  More Information

Responsible Party: Incyte Corporation Identifier: NCT01317875     History of Changes
Other Study ID Numbers: CINC424A2201 
Study First Received: March 14, 2011
Last Updated: September 23, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases processed this record on October 21, 2016