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 study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences
ClinicalTrials.gov Identifier:
NCT02124746
First received: April 24, 2014
Last updated: August 24, 2015
Last verified: August 2015
  Purpose

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.


Condition Intervention Phase
Primary Myelofibrosis
Post-Polycythemia Vera Myelofibrosis
Post-Essential Thrombocythemia Myelofibrosis
Polycythemia Vera
Essential Thrombocythemia
Drug: Momelotinib
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Gilead Sciences:

Primary Outcome Measures:
  • Adverse event profile of momelotinib as measured by incidence and severity of adverse events [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    This endpoint will measure the long-term safety and tolerability profile of momelotinib; safety information will include safety data collected after the first dose of momelotinib from the previous study.

  • Laboratory abnormality profile of momelotinib as measured by incidence and severity of clinical laboratory abnormalities [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    This endpoint will measure the long-term safety and tolerability profile of momelotinib; safety information will include safety data collected after the first dose of momelotinib from the previous study.


Secondary Outcome Measures:
  • Overall survival [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Overall survival (OS) is defined as the interval from the first dose of momelotinib on the previous study until death from any cause.

  • Progression-free survival [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]

    Progression-free survival (PFS) is defined as the interval from the first dose of momelotinib on the previous study until the first documentation of definitive progressive disease or death due to any cause.

    • Progressive disease for Cohorts 1 & 2 is defined as leukemic transformation, increase in peripheral blood blast for at least 8 weeks, or progressive splenomegaly.

  • Leukemia-free survival [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Leukemia-free survival is defined as the interval from the first dose of momelotinib on the previous study until the first documented leukemic transformation or death from any cause.

  • Rate of RBC transfusion [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Rate of red blood cell (RBC) transfusion is defined as the average number of RBC units per subject month from the first dose of momelotinib on the previous study during the study period.

  • Duration of splenic response (Cohorts 1 and 2 only) [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]

    Duration of splenic response is defined as the interval from the first onset of splenic response (in the previous study or this study) to loss of splenic response for ≥ 4 weeks or death from any cause.

    • Splenic response is defined as:

      • > 50% reduction in palpable splenomegaly of a spleen that is ≥ 10 cm below the left costal margin (LCM) at baseline or
      • a spleen becomes not palpable, that is palpable at > 5 cm below the LCM at baseline

  • Duration of transfusion independence response (Cohorts 1 and 2 only) [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]

    Duration of transfusion independence response is defined as the interval from the first onset date of transfusion independence (in the previous study or this study) to the earliest onset date of transfusion dependence or death from any cause for participants who are transfusion dependent at baseline.

    • Transfusion independence is defined as absence of RBC transfusions for ≥ 12 weeks.

  • Duration of anemia response (Cohorts 1 and 2 only) [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]

    Duration of anemia response is defined as the interval from the first onset of anemia response to the earliest date of loss of anemia response which persists for at least 4 weeks, or death from any cause

    Anemia response is defined as:

    • ≥ 2 g/dL increase in hemoglobin, if transfusion independent and with hemoglobin level of < 10 g/dL at baseline
    • Achieving transfusion independence for ≥ 12 weeks, if transfusion dependent at baseline

  • Transfusion response rate (Cohort 4 only) [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Transfusion response rate is defined as becoming not transfusion dependent for ≥ 12 weeks at any time from the first dose of MMB on the previous study until end of this study.


Estimated Enrollment: 105
Study Start Date: April 2014
Estimated Study Completion Date: June 2019
Estimated Primary Completion Date: June 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1
Participants previously enrolled in Study CCL09191E will receive momelotinib for up to 4 years.
Drug: Momelotinib
Momelotinib tablets administered orally once daily
Other Names:
  • GS-0387
  • CYT387
Experimental: Cohort 2
Participants previously enrolled in Study YM387-II-02 will receive momelotinib for up to 4 years.
Drug: Momelotinib
Momelotinib tablets administered orally once daily
Other Names:
  • GS-0387
  • CYT387
Experimental: Cohort 3

Participants 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.

Drug: Momelotinib
Momelotinib tablets administered orally once daily
Other Names:
  • GS-0387
  • CYT387
Experimental: Cohort 4
Participants previously enrolled in Study GS-US-352-1672 will receive momelotinib for up to 4 years.
Drug: Momelotinib
Momelotinib tablets administered orally once daily
Other Names:
  • GS-0387
  • CYT387

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Known hypersensitivity to momelotinib, its metabolites, or formulation excipients
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02124746

Locations
United States, Arizona
Scottsdale, Arizona, United States
United States, California
Stanford, California, United States
Whittier, California, United States
United States, Florida
Jacksonville, Florida, United States
United States, Massachusetts
Boston, Massachusetts, United States
United States, Minnesota
Rochester, Minnesota, United States
United States, Missouri
St. Louis, Missouri, United States
United States, Texas
Houston, Texas, United States
United States, Utah
Salt Lake City, Utah, United States
Australia, Victoria
Frankston, Victoria, Australia
Parkville, Victoria, Australia
Canada, Ontario
Toronto, Ontario, Canada
Canada, Quebec
Montreal, Quebec, Canada
France
La Tronche, France
Paris, France
Germany
Dresden, Germany
Minden, Germany
Sponsors and Collaborators
Gilead Sciences
Investigators
Study Director: Helen Collins, MD Gilead Sciences
  More Information

No publications provided

Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT02124746     History of Changes
Other Study ID Numbers: GS-US-352-1154, 2013-004476-36
Study First Received: April 24, 2014
Last Updated: August 24, 2015
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration
Canada: Health Canada

Keywords provided by Gilead Sciences:
Primary Myelofibrosis
Post Polycythemia Vera Myelofibrosis
Post Essential Thrombocythemia Myelofibrosis
Polycythemia Vera
Essential Thrombocythemia
blood disorders

Additional relevant MeSH terms:
Primary Myelofibrosis
Polycythemia
Polycythemia Vera
Thrombocythemia, Essential
Thrombocytosis
Blood Coagulation Disorders
Blood Platelet Disorders
Bone Marrow Diseases
Hematologic Diseases
Hemorrhagic Disorders
Myeloproliferative Disorders

ClinicalTrials.gov processed this record on August 27, 2015