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  • Cancer Type: Malignant Hematology
  • Study Type: Treatment
  • NCT#: NCT04576156
  • Phase: Phase III
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  • Overview

    Study Title:

    A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK)-Inhibitor


    The purpose of the study is to evaluate the overall survival of participants treated with imetelstat compared to best available therapy with intermediate-2 or high-risk Myelofibrosis (MF) who are relapsed/refractory to Janus Kinase (JAK)-Inhibitor treatment.


    The primary objective of this study is to compare the OS of participants, treated with imetelstat versus best available therapy (BAT), with intermediate-2 or high-risk MF whose disease is refractory to JAK-inhibitor treatment. The primary endpoint of OS is defined as the time interval from randomization date to date of death from any cause.

  • Treatments


    Therapy (NOS)


    Imetelstat (GRN163L)

  • Inclusion Criteria

      Inclusion Criteria:
    • Diagnosis of primary myelofibrosis according to the revised World Health Organization criteria or post-essential thrombocythemia-MF or post-polycythemia vera-MF according to the IWG-MRT criteria
    • Dynamic International Prognostic Scoring System intermediate-2 or high-risk MF
    • Relapsed/refractory to JAK-inhibitor treatment as defined in either inclusion (i), (ii) or (iii) and not eligible for allogeneic stem cell transplantation (ASCT) at screening: (i) Treatment with JAK-inhibitor for >= 6 months duration, including at least 2 months at an optimal dose as assessed by the investigator for that participant and at least one of the following: (a) no decrease in spleen volume (= 3 months duration with maximal doses (e.g., 20-25 mg twice daily ruxolitinib) for that participant and no decrease in spleen volume/size or symptoms as defined in inclusion criterion (i [a, b, or c]). (iii) Following maximum tolerated doses of JAK inhibitor therapy for ≥3 months duration, having documented relapsed disease defined as either (a) Increase in spleen volume from time of best response by 25% measured by MRI or CT, or (b) Increase in spleen size by palpation, CT, or ultrasound (b.i) For splenomegaly of 5-10 cm at the start of JAK inhibitor treatment, at least 100% increase in palpable spleen size from time of best response;(b.ii) For splenomegaly of > 10 cm at the start of JAK inhibitor treatment, at least 50% increase in palpable spleen size from time of best response; AND not a candidate for further JAK inhibitor at screening per investigator.
    • Measurable splenomegaly demonstrated by a palpable spleen measuring >= 5 cm below the left costal margin or a spleen volume >= 450 cm^3 by MRI or CT
    • Active symptoms of MF on the MFSAF v4.0 demonstrated by a symptom score of at least 5 points (on a 0 to 10 scale)
    • Hematology laboratory test values within the protocol defined limits
    • Biochemical laboratory test values must be within protocol defined limits
    • Eastern Cooperative Oncology Group Performance Status score of 0, 1, or 2
    • Participants should follow protocol defined contraceptives procedures
    • A woman of childbearing potential must have a negative serum or urine pregnancy test at screening
    • Other criteria may apply
  • Exclusion Criteria

      Exclusion Criteria:
    • Peripheral blood blast count of >= 10% or bone marrow blast count of >=10%
    • Known allergies, hypersensitivity, or intolerance to imetelstat or its excipients
    • Prior treatment with imetelstat
    • Any chemotherapy or MF directed therapy, including investigational drug regardless of class or mechanism of action, immunomodulatory or immunosuppressive therapy, corticosteroids greater than 30 mg/day prednisone or equivalent, and JAK-inhibitor treatment less than equal to 14 days prior to randomization
    • Diagnosis or treatment for malignancy other than MF except: (a) Malignancy treated with curative intent and with no known active disease present for >= 3 years before randomization (b) Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease (c) Adequately treated cervical carcinoma in situ without evidence of disease
    • Known history of human immunodeficiency virus or any uncontrolled active systemic infection requiring IV antibiotics
    • Active systemic hepatitis infection requiring treatment (carriers of hepatitis virus are permitted to enter the study), or any known acute or chronic liver disease requiring treatment unless related to underlying hepatosplenomegaly due to MF
    • Major surgery within 28 days prior to randomization
    • Any life-threatening illness (e.g., coronavirus disease-2019), medical condition, or organ system dysfunction which, in the investigator's opinion, could compromise the participant's safety, interfere with the imetelstat metabolism, or put the study outcomes at undue risk
    • Other criteria may apply

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