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

Primary Purpose

Polycythemia Vera, Essential Thrombocythemia

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Momelotinib
Sponsored by
Sierra Oncology LLC - a GSK company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycythemia Vera focused on measuring Polycythemia, Polycythemia Vera, Essential Thrombocythemia, Thrombocytosis, Myeloproliferative Disorders, Bone Marrow Diseases, Hematologic Diseases, Blood Coagulation Disorders, Blood Platelet Disorders, Hemorrhagic Disorders

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Momelotinib 100 mg PV

Momelotinib 200 mg PV

Momelotinib 100 mg ET

Momelotinib 200 mg ET

Arm Description

Participants with polycythemia vera will receive 100 mg of momelotinib.

Participants with polycythemia vera will receive 200 mg of momelotinib.

Participants with essential thrombocythemia will receive 100 mg of momelotinib.

Participants with essential thrombocythemia will receive 200 mg of momelotinib.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Confirmed overall response rate
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.
Proportion of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks
Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
Proportion of participants with platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks
Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 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

Full Information

First Posted
November 25, 2013
Last Updated
April 14, 2020
Sponsor
Sierra Oncology LLC - a GSK company
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1. Study Identification

Unique Protocol Identification Number
NCT01998828
Brief Title
Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia
Official Title
A Phase 2, Open-label, Randomized Study to Evaluate the Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia
Study Type
Interventional

2. Study Status

Record Verification Date
April 2020
Overall Recruitment Status
Terminated
Study Start Date
February 19, 2014 (Actual)
Primary Completion Date
March 17, 2015 (Actual)
Study Completion Date
May 7, 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sierra Oncology LLC - a GSK company

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycythemia Vera, Essential Thrombocythemia
Keywords
Polycythemia, Polycythemia Vera, Essential Thrombocythemia, Thrombocytosis, Myeloproliferative Disorders, Bone Marrow Diseases, Hematologic Diseases, Blood Coagulation Disorders, Blood Platelet Disorders, Hemorrhagic Disorders

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
39 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Momelotinib 100 mg PV
Arm Type
Experimental
Arm Description
Participants with polycythemia vera will receive 100 mg of momelotinib.
Arm Title
Momelotinib 200 mg PV
Arm Type
Experimental
Arm Description
Participants with polycythemia vera will receive 200 mg of momelotinib.
Arm Title
Momelotinib 100 mg ET
Arm Type
Experimental
Arm Description
Participants with essential thrombocythemia will receive 100 mg of momelotinib.
Arm Title
Momelotinib 200 mg ET
Arm Type
Experimental
Arm Description
Participants with essential thrombocythemia will receive 200 mg of momelotinib.
Intervention Type
Drug
Intervention Name(s)
Momelotinib
Other Intervention Name(s)
GS-0387, CYT387
Intervention Description
Momelotinib tablet administered orally once daily
Primary Outcome Measure Information:
Title
Overall response rate
Description
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
Time Frame
Up to 24 weeks
Secondary Outcome Measure Information:
Title
Confirmed overall response rate
Description
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.
Time Frame
Up to 24 weeks
Title
Proportion of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks
Time Frame
Up to 24 weeks
Title
Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
Time Frame
Up to 24 weeks
Title
Proportion of participants with platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks
Time Frame
Up to 24 weeks
Title
Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 weeks
Time Frame
Up to 24 weeks
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peter Lee, MD, PhD
Organizational Affiliation
Gilead Sciences
Official's Role
Study Director
Facility Information:
City
Scottsdale
State/Province
Arizona
Country
United States
City
Whittier
State/Province
California
Country
United States
City
Tupelo
State/Province
Mississippi
Country
United States
City
Saint Louis
State/Province
Missouri
Country
United States
City
Houston
State/Province
Texas
Country
United States
City
Frankston
State/Province
Victoria
Country
Australia
City
Parkville
State/Province
Victoria
Country
Australia
City
Vancouver
State/Province
British Columbia
Country
Canada
City
Toronto
State/Province
Ontario
Country
Canada
City
Montreal
State/Province
Quebec
Country
Canada
City
La Tronche
Country
France
City
Nantes Cedex 1
Country
France
City
Paris
Country
France
City
Dresden
Country
Germany
City
Minden
Country
Germany

12. IPD Sharing Statement

Citations:
PubMed Identifier
28622623
Citation
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.
Results Reference
derived

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

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