NCT01998828

Brief Summary

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
39

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Feb 2014

Shorter than P25 for phase_2

Geographic Reach
5 countries

15 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 25, 2013

Completed
7 days until next milestone

First Posted

Study publicly available on registry

December 2, 2013

Completed
3 months until next milestone

Study Start

First participant enrolled

February 19, 2014

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 17, 2015

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 7, 2015

Completed
Last Updated

April 20, 2020

Status Verified

April 1, 2020

Enrollment Period

1.1 years

First QC Date

November 25, 2013

Last Update Submit

April 14, 2020

Conditions

Polycythemia VeraEssential Thrombocythemia

Keywords

PolycythemiaPolycythemia VeraEssential ThrombocythemiaThrombocytosisMyeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesBlood Coagulation DisordersBlood Platelet DisordersHemorrhagic Disorders

Outcome Measures

Primary Outcomes (1)

  • Overall response rate

    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 Outcomes (6)

  • Confirmed overall response rate

    Up to 24 weeks

  • Proportion of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks

    Up to 24 weeks

  • Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks

    Up to 24 weeks

  • Proportion of participants with platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks

    Up to 24 weeks

  • Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 weeks

    Up to 24 weeks

  • +1 more secondary outcomes

Study Arms (4)

Momelotinib 100 mg PV

EXPERIMENTAL

Participants with polycythemia vera will receive 100 mg of momelotinib.

Drug: Momelotinib

Momelotinib 200 mg PV

EXPERIMENTAL

Participants with polycythemia vera will receive 200 mg of momelotinib.

Drug: Momelotinib

Momelotinib 100 mg ET

EXPERIMENTAL

Participants with essential thrombocythemia will receive 100 mg of momelotinib.

Drug: Momelotinib

Momelotinib 200 mg ET

EXPERIMENTAL

Participants with essential thrombocythemia will receive 200 mg of momelotinib.

Drug: Momelotinib

Interventions

MomelotinibDRUG

Momelotinib tablet administered orally once daily

Also known as: GS-0387, CYT387
Momelotinib 100 mg ETMomelotinib 100 mg PVMomelotinib 200 mg ETMomelotinib 200 mg PV

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Unknown Facility

Scottsdale, Arizona, United States

Location

Unknown Facility

Whittier, California, United States

Location

Unknown Facility

Tupelo, Mississippi, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Frankston, Victoria, Australia

Location

Unknown Facility

Parkville, Victoria, Australia

Location

Unknown Facility

Vancouver, British Columbia, Canada

Location

Unknown Facility

Toronto, Ontario, Canada

Location

Unknown Facility

Montreal, Quebec, Canada

Location

Unknown Facility

La Tronche, France

Location

Unknown Facility

Nantes, France

Location

Unknown Facility

Paris, France

Location

Unknown Facility

Dresden, Germany

Location

Unknown Facility

Minden, Germany

Location

Related Publications (1)

  • Verstovsek S, Courby S, Griesshammer M, Mesa RA, Brachmann CB, Kawashima J, Maltzman JD, Shao L, Xin Y, Huang D, Bajel A. A phase 2 study of momelotinib, a potent JAK1 and JAK2 inhibitor, in patients with polycythemia vera or essential thrombocythemia. Leuk Res. 2017 Sep;60:11-17. doi: 10.1016/j.leukres.2017.05.002. Epub 2017 May 30.

    PMID: 28622623DERIVED

MeSH Terms

Conditions

Polycythemia VeraThrombocythemia, EssentialPolycythemiaThrombocytosisMyeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesBlood Coagulation DisordersBlood Platelet DisordersHemorrhagic Disorders

Interventions

N-(cyanomethyl)-4-(2-((4-(4-morpholinyl)phenyl)amino)-4-pyrimidinyl)benzamide

Condition Hierarchy (Ancestors)

Bone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsHemic and Lymphatic Diseases

Study Officials

  • Peter Lee, MD, PhD

    Gilead Sciences

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 25, 2013

First Posted

December 2, 2013

Study Start

February 19, 2014

Primary Completion

March 17, 2015

Study Completion

May 7, 2015

Last Updated

April 20, 2020

Record last verified: 2020-04

Locations

CA(3)AU(2)FR(3)US(5)DE(2)