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Primary MyelofibrosisPhase 2NCT02124746

Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Polycythemia Vera or Essential Thrombocythemia

A Phase 2 study testing Momelotinib in people with Primary Myelofibrosis and Post-Polycythemia Vera Myelofibrosis, run by Sierra Oncology LLC - a GSK company at 22 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This open-label study is to determine the long-term safety and tolerability of momelotinib in previously enrolled study participants with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), post-essential thrombocythemia myelofibrosis (post-ET MF), polycythemia vera (PV), or essential… Read more about this study

Who is it for?
Ages 18 years and older
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
87 participants
Where
22 sitesin 5 countries
Study dates
30 Apr 2014to 6 Dec 2018
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Currently enrolled in study CCL09101E, or YM387-II-02, or successfully completed 24 weeks of study GS-US-352-1672
  • Able to comprehend and willing to sign informed consent form

Who cannot join

  • Known hypersensitivity to momelotinib, its metabolites, or formulation excipients
  • Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

22 sites in 5 countries.

22 sites in 5 countries

United States15 sites
  • Study site

    Ann Arbor, Michigan

  • Study site

    Baltimore, Maryland

  • Study site

    Boston, Massachusetts

  • Study site

    Cleveland, Ohio

  • Study site

    Houston, Texas

  • Study site

    Jacksonville, Florida

  • Study site

    New York, New York

  • Study site

    Orange, California

  • Study site

    Rochester, Minnesota

  • Study site

    Salt Lake City, Utah

  • Study site

    Scottsdale, Arizona

  • Study site

    St Louis, Missouri

  • Study site

    Stanford, California

  • Study site

    The Bronx, New York

  • Study site

    Whittier, California

Other trials in United States

Australia2 sites
  • Study site

    Frankston, Victoria

  • Study site

    Parkville, Victoria

Other trials in Australia

Canada2 sites
  • Study site

    Montreal, Quebec

  • Study site

    Toronto, Ontario

Other trials in Canada

France2 sites
  • Study site

    La Tronche

  • Study site

    Paris

Other trials in France

Germany1 site
  • Study site

    Minden

Other trials in Germany

About this study

Official title: Open-label Study to Assess the Long-term Safety and Efficacy of Momelotinib in Subjects With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis, Polycythemia Vera or Essential Thrombocythemia

This open-label study is to determine the long-term safety and tolerability of momelotinib in previously enrolled study participants with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), post-essential thrombocythemia myelofibrosis (post-ET MF), polycythemia vera (PV), or essential thrombocythemia (ET), who have tolerated and achieved stable disease or better with momelotinib treatment while enrolled in a previous clinical trial.

Being tested
Momelotinib
Keywords
Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis, Polycythemia Vera, Essential Thrombocythemia, blood disorders
Medical subject headings
Primary Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential, Hematologic Diseases

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 tablets administered orally once daily

Study groups in NCT02124746
Study groupRoleWhat they receive
Cohort 1ExperimentalParticipants previously enrolled in Study CCL09191E will receive momelotinib for approximately 4 years.
Receives: Drug: Momelotinib
Cohort 2ExperimentalParticipants previously enrolled in Study YM387-II-02 will receive momelotinib for approximately 4 years.
Receives: Drug: Momelotinib
Cohort 3ExperimentalParticipants previously enrolled in Study GS-US-354-0101 will receive momelotinib for up to 4 years. Cohort 3 was closed and all enrolled participants were discontinued from this study because parent Study GS-US-354-0101 was terminated.
Receives: Drug: Momelotinib
Cohort 4ExperimentalParticipants previously enrolled in Study GS-US-352-1672 will receive momelotinib for approximately 4 years.
Receives: Drug: Momelotinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Long Term Safety and Tolerability as Measured by the Incidence and Severity of Adverse Events and Clinical Laboratory Abnormalities

    Long-term safety and tolerability profile of momelotinib based on safety data (adverse events and selected hematology and chemistry laboratory parameters) collected after the first dose of momelotinib in the parent study.

    Measured: From the first dose of momelotinib in the parent study to 30 days following permanent discontinuation of momelotinib in Study GS-US-352-1154.

Secondary outcomes (10)
  1. Splenic Response Rate

    The number of subjects achieving a spleen response, defined as a reduction of 50% or more in palpable splenomegaly of a spleen that was at least 10 cm below the LCM at baseline, or a spleen that was palpable at > 5 cm and < 10 cm below the LCM at baseline becoming not palpable for at least 56 days, using baseline of the parent study as the reference.

    Measured: From baseline in the parent study until the last spleen assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.

  2. Duration of Splenic Response

    The interval from the first onset of splenic response (in the parent study or Study GS-US-352-1154) to the earliest date of loss of splenic response. Loss of response was defined as the reduction of splenomegaly by < 50% among responders (with splenomegaly ≥ 10 cm below the LCM at baseline) that lasts ≥ 56 days, or the recurrence of > 0 cm splenomegaly among responders (with splenomegaly > 5 and < 10 cm at baseline) that lasts ≥ 56 days.

    Duration of splenic response was measured by descriptive statistics. Data from responders who maintained their response was censored at the last assessment date.

    Measured: From baseline in the parent study until the last spleen assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.

  3. Transfusion Independence Response Rate

    The number of transfusion dependent subjects at entry to a parent study who became transfusion-independent for ≥ 12 weeks at any time from the first dose of momelotinib in the parent study until the end of Study GS-US-352-1154.

    Measured: From baseline in the parent study until the last assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.

  4. Duration of Transfusion Independence Response

    The interval from the first onset date of transfusion independence (in the parent study or Study GS-US-352-1154) to the earliest date of loss of response for participants who are transfusion dependent at baseline in the parent study. Loss of TI response was defined as receiving an RBC transfusion after achieving a TI response.

    Duration of transfusion independence response was measured by descriptive statistics. Data from responders who maintained their response was censored at the last assessment date.

    Measured: From baseline in the parent study until the last assessment date in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.

  5. Anemia Response Rate

    The number of subjects achieving an anemia response, defined as:

    • Achieving transfusion independence for ≥ 12 weeks, for subjects who were transfusion-dependent at baseline in the parent study, or
    • Having ≥ 2 g/dL increase in Hgb from baseline for ≥ 12 weeks, for subjects with Hgb < 10 g/dL at baseline in the parent study who were not transfusion-dependent (Cohort 1) or who were transfusion-independent (Cohort 2).

    Measured: From baseline in the parent study until the last assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.

  6. Duration of Anemia Response

    The interval from the first onset of anemia response (in the parent study or Study GS-US-352-1154) to the earliest date of loss of anemia response. Loss of anemia response was defined as having any RBC transfusion after achieving an anemia response.

    Duration of anemia response was measured by descriptive statistics. Data from responders who maintained their response was censored at the last assessment date.

    Measured: From baseline in the parent study until the last assessment in Study GS-US-352-1154, up to 30 days following permanent discontinuation of momelotinib.

  7. Rate of RBC Transfusion

    The average number of RBC units per subject month during the parent study and/or Study GS-US-352-1154.

    Measured: From the first dose of momelotinib in the parent study until the last dose of momelotinib in Study GS-US-352-1154.

  8. Overall Survival

    The interval from the first dose of momelotinib in the parent study until death from any cause.

    Overall survival was analyzed using the Kaplan-Meier method. Data from subjects who were lost to follow-up or remained alive until the end of the study were censored at the date of last contact or last response assessment.

    Measured: From baseline in the parent study until the date of last contact or last response assessment, up to 30 days following permanent discontinuation of momelotinib.

  9. Progression-Free Survival

    The interval from the first dose of momelotinib in the parent study until the first documentation of definitive progressive disease as defined in 2006 IWG-MRT or death due to any cause.

    Subjects who were free of progression were censored at the last assessment date.

    Measured: From baseline in the parent study until the last response assessment, up to 30 days following permanent discontinuation of momelotinib.

  10. Leukemia-Free Survival

    The interval from the first dose of momelotinib in the parent study until the first documented leukemic transformation or death from any cause. Leukemic transformation was documented in the adverse event electronic case report form.

    Leukemia-free survival was analyzed using the Kaplan-Meier method. Subjects who were free of leukemia transformation were censored at the last assessment date.

    Measured: From baseline in the parent study until the date of the last assessment, up to 30 days following permanent discontinuation of momelotinib.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted28 Apr 2014 (estimated)
  2. Study start30 Apr 2014
  3. Primary completion6 Dec 2018
  4. Study completion6 Dec 2018

Resources

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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
87 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02124746
Sponsor's protocol ID
GS-US-352-1154
Other IDs
2013-004476-36
Status last verified
Jun 2023
First submitted
24 Apr 2014
Last updated
18 Jun 2023
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Momelotinib in people with Primary Myelofibrosis and Post-Polycythemia Vera Myelofibrosis, run by Sierra Oncology LLC - a GSK company at 22 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 2 inclusion and 2 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: finished, as of the sponsor's last update on 18 Jun 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 22 sites in United States, Australia, Canada, France, 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?

Yes. Results are posted in the registry (first posted 5 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Long Term Safety and Tolerability as Measured by the Incidence and Severity of Adverse Events and Clinical Laboratory Abnormalities (time frame: From the first dose of momelotinib in the parent study to 30 days following permanent discontinuation of momelotinib in Study GS-US-352-1154.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
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 18 Jun 2023.

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