PF-04449913 Single-Agent In Patients with Myelofibrosis Previously Treated with a Janus Kinase Inhibitor

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Scottsdale/Phoenix, Arizona: 14-004331
    • Jacksonville, Florida: 14-004331
    NCT ID: NCT02226172
    Sponsor Protocol Number: B1371013

About this study

A lead-in cohort of PF-04449913 100mg daily dose will be evaluated in symptomatic primary or secondary myelofibrosis patients previously treated with 1 or more licensed or experimental Janus kinase inhibitors. Following the lead-in, a phase 2, randomized, double blind, 2 arm study of oral single agent PF 04449913 versus placebo will be tested in patients resistant or intolerant to ruxolitinib.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Diagnosis of primary or secondary myelofibrosis as per World Health Organization 2008 criteria.
  • Prior treatment with 1 or more JAK inhibitors (must meet one of the following criteria):
    1. Previous treatment with 1 or more JAK inhibitors (licensed or experimental) for a minimum duration of at least 4 weeks and failure to achieve or sustain adequate symptomatic control and/or achieve or sustain an adequate reduction of splenomegaly (Investigator's judgment);
    2. JAKi therapy discontinuation for unacceptable toxicity irrespective of the duration of therapy.
  • Spleen at least 5 cm below the inferior left costal margin as measured by manual palpation.
  • Symptomatic myelofibrosis
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2

Exclusion Criteria:

  • Previous treatment with a licensed or experimental smoothened inhibitor.
  • Other anti cancer therapy up to 14 days prior to enrollment, with the exception of hydroxyurea, which can be given up to 7 days prior to enrollment.
  • Splenic irradiation within 3 months prior to enrollment.
  • History of congenital long QT syndrome, or a baseline =>470 msec QTcF abnormality (average of the triplicate reading).

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Ruben Mesa, M.D.

Closed for enrollment

Contact information:

Research Information Center

800-664-4542

Jacksonville, Fla.

Mayo Clinic principal investigator

Ruben Mesa, M.D.

Closed for enrollment

Contact information:

Research Information Center

800-664-4542

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CLS-20143570

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