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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: February 28, 2017
Last verified: February 2017
  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
Intervention Model: Parallel Assignment
Masking: No masking
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 ]
    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 ]
    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 ]
    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 ]

    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 ]
    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 ]
    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 ]

    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 ]

    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 ]

    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 ]
    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
Actual Study Start Date: April 2014
Estimated Study Completion Date: August 2021
Estimated Primary Completion Date: August 2021 (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   (Adult, Senior)
Sexes Eligible for Study:   All
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

  Show 21 Study Locations
Sponsors and Collaborators
Gilead Sciences
Investigators
Study Director: Gilead Study Director Gilead Sciences
  More Information

Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT02124746     History of Changes
Other Study ID Numbers: GS-US-352-1154
2013-004476-36 ( EudraCT Number )
Study First Received: April 24, 2014
Last Updated: February 28, 2017

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
Thrombocytosis
Thrombocythemia, Essential
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Blood Platelet Disorders
Blood Coagulation Disorders
Hemorrhagic Disorders

ClinicalTrials.gov processed this record on April 28, 2017