Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia

April 14, 2020 updated by: Sierra Oncology, Inc.

A Phase 2, Open-label, Randomized Study to Evaluate the Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia

This open-label study is to determine the safety and efficacy of momelotinib in participants with either polycythemia vera (PV) or essential thrombocythemia (ET) who have not yet received treatment with a Janus kinase (JAK) inhibitor.

Study Overview

Status

Terminated

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

39

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Victoria
      • Frankston, Victoria, Australia
      • Parkville, Victoria, Australia
    • British Columbia
      • Vancouver, British Columbia, Canada
    • Ontario
      • Toronto, Ontario, Canada
    • Quebec
      • Montreal, Quebec, Canada
      • La Tronche, France
      • Nantes Cedex 1, France
      • Paris, France
      • Dresden, Germany
      • Minden, Germany
    • Arizona
      • Scottsdale, Arizona, United States
    • California
      • Whittier, California, United States
    • Mississippi
      • Tupelo, Mississippi, United States
    • Missouri
      • Saint Louis, Missouri, United States
    • Texas
      • Houston, Texas, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Diagnosis of either PV or ET as defined by the 2008 World Health Organization (WHO) Diagnostic Criteria
  • Requires treatment for PV or ET, in the opinion of the study investigator
  • Intolerant of, resistant to, or refuses current or available treatment for PV or ET
  • Direct bilirubin ≤ 2.0 x upper limit of the normal range (ULN)
  • Aspartate transaminase (AST) and alanine transaminase (ALT) ≤ 3 x ULN
  • Calculated creatinine clearance (CrCl) of ≥ 45 mL/min
  • Life expectancy > 24 weeks
  • Male subjects and female subjects of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception
  • Females who are nursing must agree to discontinue nursing before the first dose of study drug
  • Able to comprehend and willing to sign informed consent form

Exclusion Criteria:

  • Prior splenectomy
  • Uncontrolled intercurrent illness, per protocol
  • Known positive status for human immunodeficiency virus (HIV)
  • Chronic active or acute viral hepatitis A, B, or C infection, or hepatitis B or C carrier
  • Myeloproliferative neoplasm-directed therapy, other than aspirin, hydroxyurea, anagrelide, and/or phlebotomy, within 21 days prior to the first dose of study drug
  • Anagrelide within 7 days prior to the first dose of study drug
  • Presence of peripheral neuropathy ≥ Grade 2
  • Unwilling or unable to take oral medication
  • Prior use of a JAK1 or JAK2 inhibitor
  • Use of strong CYP3A4 inducers within 1 week prior to the first dose of study drug
  • QTc interval > 450 msec, unless attributed to bundle branch block

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Momelotinib 100 mg PV
Participants with polycythemia vera will receive 100 mg of momelotinib.
Momelotinib tablet administered orally once daily
Other Names:
  • GS-0387
  • CYT387
Experimental: Momelotinib 200 mg PV
Participants with polycythemia vera will receive 200 mg of momelotinib.
Momelotinib tablet administered orally once daily
Other Names:
  • GS-0387
  • CYT387
Experimental: Momelotinib 100 mg ET
Participants with essential thrombocythemia will receive 100 mg of momelotinib.
Momelotinib tablet administered orally once daily
Other Names:
  • GS-0387
  • CYT387
Experimental: Momelotinib 200 mg ET
Participants with essential thrombocythemia will receive 200 mg of momelotinib.
Momelotinib tablet administered orally once daily
Other Names:
  • GS-0387
  • CYT387

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall response rate
Time Frame: Up to 24 weeks

For the PV Cohort, overall response rate (ORR) is defined as the proportion of participants with all of the following at some point during the treatment period:

  • Hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks
  • White blood cell (WBC) count < 10 x 10^9/L that lasts at least 4 weeks
  • Platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks
  • Resolution of palpable splenomegaly that lasts at least 4 weeks

For the ET Cohort, overall response rate is defined as the proportion of participants with all of the following at some point during the treatment period:

  • WBC count < 10 x 10^9/L that lasts at least 4 weeks
  • Platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks
  • Resolution of palpable splenomegaly that lasts at least 4 weeks
Up to 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Confirmed overall response rate
Time Frame: Up to 24 weeks
Confirmed overall response rate is defined as the proportion of participants who meet all the criteria listed for the primary endpoints of PV or ET, sustained for at least 12 weeks.
Up to 24 weeks
Proportion of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks
Time Frame: Up to 24 weeks
Up to 24 weeks
Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
Time Frame: Up to 24 weeks
Up to 24 weeks
Proportion of participants with platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks
Time Frame: Up to 24 weeks
Up to 24 weeks
Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 weeks
Time Frame: Up to 24 weeks
Up to 24 weeks
Proportion of participants with ≥ 10 point decrease in modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPNSAF TSS) compared to baseline that lasts at least 12 weeks
Time Frame: Up to 24 weeks
Up to 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Study Director: Peter Lee, MD, PhD, Gilead Sciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 19, 2014

Primary Completion (Actual)

March 17, 2015

Study Completion (Actual)

May 7, 2015

Study Registration Dates

First Submitted

November 25, 2013

First Submitted That Met QC Criteria

November 25, 2013

First Posted (Estimate)

December 2, 2013

Study Record Updates

Last Update Posted (Actual)

April 20, 2020

Last Update Submitted That Met QC Criteria

April 14, 2020

Last Verified

April 1, 2020

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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