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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01998828




Registration number
NCT01998828
Ethics application status
Date submitted
25/11/2013
Date registered
2/12/2013
Date last updated
20/04/2020

Titles & IDs
Public title
Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia
Scientific title
A Phase 2, Open-label, Randomized Study to Evaluate the Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia
Secondary ID [1] 0 0
2013-004105-11
Secondary ID [2] 0 0
GS-US-354-0101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Polycythemia Vera 0 0
Essential Thrombocythemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Momelotinib

Experimental: Momelotinib 100 mg PV - Participants with polycythemia vera will receive 100 mg of momelotinib.

Experimental: Momelotinib 200 mg PV - Participants with polycythemia vera will receive 200 mg of momelotinib.

Experimental: Momelotinib 100 mg ET - Participants with essential thrombocythemia will receive 100 mg of momelotinib.

Experimental: Momelotinib 200 mg ET - Participants with essential thrombocythemia will receive 200 mg of momelotinib.


Treatment: Drugs: Momelotinib
Momelotinib tablet administered orally once daily

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to 24 weeks
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
Up to 24 weeks
Secondary outcome [2] 0 0
Proportion of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks
Timepoint [2] 0 0
Up to 24 weeks
Secondary outcome [3] 0 0
Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
Timepoint [3] 0 0
Up to 24 weeks
Secondary outcome [4] 0 0
Proportion of participants with platelet count = 400 x 10^9/L that lasts at least 4 weeks
Timepoint [4] 0 0
Up to 24 weeks
Secondary outcome [5] 0 0
Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 weeks
Timepoint [5] 0 0
Up to 24 weeks
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
Up to 24 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
- Frankston
Recruitment hospital [2] 0 0
- Parkville
Recruitment postcode(s) [1] 0 0
- Frankston
Recruitment postcode(s) [2] 0 0
- Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Mississippi
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
Texas
Country [6] 0 0
Canada
State/province [6] 0 0
British Columbia
Country [7] 0 0
Canada
State/province [7] 0 0
Ontario
Country [8] 0 0
Canada
State/province [8] 0 0
Quebec
Country [9] 0 0
France
State/province [9] 0 0
La Tronche
Country [10] 0 0
France
State/province [10] 0 0
Nantes Cedex 1
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
Germany
State/province [12] 0 0
Dresden
Country [13] 0 0
Germany
State/province [13] 0 0
Minden

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Sierra Oncology, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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 website
https://clinicaltrials.gov/show/NCT01998828
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Peter Lee, MD, PhD
Address 0 0
Gilead Sciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01998828