Polycythemia VeraPhase 2NCT01998828
Safety and Efficacy of Momelotinib in Subjects With Polycythemia Vera or Essential Thrombocythemia
A Phase 2 study testing Momelotinib in people with Polycythemia Vera and Essential Thrombocythemia, run by Sierra Oncology LLC - a GSK company at 15 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera, Essential Thrombocythemia
- What is being tested?
- Momelotinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 15 sitesin 5 countries
- Study dates
- 19 Feb 2014to 7 May 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
15 sites in 5 countries.
15 sites in 5 countries
United States5 sites
Study site
Houston, Texas
Study site
Scottsdale, Arizona
Study site
St Louis, Missouri
Study site
Tupelo, Mississippi
Study site
Whittier, California
Canada3 sites
Study site
Montreal, Quebec
Study site
Toronto, Ontario
Study site
Vancouver, British Columbia
Australia2 sites
Study site
Frankston, Victoria
Study site
Parkville, Victoria
About this study
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
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.
- Conditions
- Polycythemia Vera, Essential Thrombocythemia
- Being tested
- Momelotinib
- Keywords
- Polycythemia, Polycythemia Vera, Essential Thrombocythemia, Thrombocytosis, Myeloproliferative Disorders, Bone Marrow Diseases, Hematologic Diseases, Blood Coagulation Disorders, Blood Platelet Disorders, Hemorrhagic Disorders
- Medical subject headings
- Polycythemia Vera, Thrombocythemia, Essential, Polycythemia, Thrombocytosis, Myeloproliferative Disorders, Bone Marrow Diseases, Hematologic Diseases, Blood Coagulation Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MomelotinibDrug
Also called GS-0387, CYT387
Momelotinib tablet administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| Momelotinib 100 mg PV | Experimental | Participants with polycythemia vera will receive 100 mg of momelotinib. Receives: Drug: Momelotinib |
| Momelotinib 200 mg PV | Experimental | Participants with polycythemia vera will receive 200 mg of momelotinib. Receives: Drug: Momelotinib |
| Momelotinib 100 mg ET | Experimental | Participants with essential thrombocythemia will receive 100 mg of momelotinib. Receives: Drug: Momelotinib |
| Momelotinib 200 mg ET | Experimental | Participants with essential thrombocythemia will receive 200 mg of momelotinib. Receives: Drug: Momelotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 24 weeks
Secondary outcomes (6)
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.
Measured: Up to 24 weeks
Proportion of participants with hematocrit < 45% in the absence of phlebotomy that lasts at least 4 weeks
Measured: Up to 24 weeks
Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
Measured: Up to 24 weeks
Proportion of participants with platelet count ≤ 400 x 10^9/L that lasts at least 4 weeks
Measured: Up to 24 weeks
Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 weeks
Measured: 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
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2013 (estimated)
- Study start19 Feb 2014
- Primary completion17 Mar 2015
- Study completion7 May 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Polycythemia VeraStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sierra Oncology LLC - a GSK company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Peter Lee, MD, PhD, Study director, Gilead Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01998828
- Sponsor's protocol ID
- GS-US-354-0101
- Other IDs
- 2013-004105-11
- Status last verified
- Apr 2020
- First submitted
- 25 Nov 2013
- Last updated
- 20 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 28622623
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01998828
- Acronym / sponsor ID
- GS-US-354-0101
- Conditions
- Polycythemia Vera, Essential Thrombocythemia
- Keywords
- Polycythemia, Polycythemia Vera, Essential Thrombocythemia, Thrombocytosis, Myeloproliferative Disorders, Bone Marrow Diseases, Hematologic Diseases, Blood Coagulation Disorders
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Sierra Oncology LLC - a GSK companyIndustry (pharma, biotech, devices)
- Study start
- 19 Feb 2014 (actual)
- Primary completion
- 17 Mar 2015 (actual)
- Study completion
- 7 May 2015 (actual)
- First posted
- 2 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Momelotinib 100 mg PV | Experimental | Drug: MomelotinibParticipants with polycythemia vera will receive 100 mg of momelotinib. |
| Momelotinib 200 mg PV | Experimental | Drug: MomelotinibParticipants with polycythemia vera will receive 200 mg of momelotinib. |
| Momelotinib 100 mg ET | Experimental | Drug: MomelotinibParticipants with essential thrombocythemia will receive 100 mg of momelotinib. |
| Momelotinib 200 mg ET | Experimental | Drug: MomelotinibParticipants with essential thrombocythemia will receive 200 mg of momelotinib. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MomelotinibAlso: GS-0387, CYT387Momelotinib tablet administered orally once daily | Drug | Momelotinib 100 mg ET, Momelotinib 100 mg PV, Momelotinib 200 mg ET, Momelotinib 200 mg PV |
Primary outcome
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
6 secondary outcomes
Confirmed overall response rate
Time frame: 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
Proportion of participants with WBC < 10 x 10^9/L that lasts at least 4 weeks
Time frame: 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
Proportion of participants with resolution of palpable splenomegaly that lasts at least 4 weeks
Time frame: 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
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2020.
- Derived from this study: PubMed 28622623Verstovsek 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter Lee, MD, PhD study director
Gilead Sciences
15 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Momelotinib in people with Polycythemia Vera and Essential Thrombocythemia, run by Sierra Oncology LLC - a GSK company at 15 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Canada, France, Australia, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early. Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall response rate (time frame: Up to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01998828, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Apr 2020.