Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis
Condition: Primary Myelofibrosis; Secondary Myelofibrosis; Myeloid Malignancies
MeSH terms: Neoplasms; Neoplasm Metastasis; Primary Myelofibrosis
Intervention: Imetelstat
Intervention description: Imetelstat sodium administered as IV over 2 hours with treatment as long as participants derive clinical benefit or until end of study.
Intervention MeSH terms: Imetelstat; Motesanib diphosphate
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 80.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Myelofibrosis; Myelodysplastic Syndromes; Myeloproliferative Neoplasm; Imetelstat; GRN163L

Summary:

      This pilot clinical trial studies how well imetelstat sodium works in treating participants
      with primary or secondary myelofibrosis and other myeloid malignancies. Imetelstat sodium may
      stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of one of the following:

          -  Primary myelofibrosis (PMF) per the revised World Health Organization (WHO) criteria.

          -  Post-polycythemia vera/essential thrombocythemia myelofibrosis (Post-ET/PV MF) per the
             International Working Group for Myeloproliferative Neoplasms Research and Treatment
             (IWG-MRT) criteria.

          -  High-risk or Intermediate-2 risk MF (as defined by the Dynamic International
             Prognostic Scoring System [DIPSS-plus]).

          -  Life expectancy of greater than or equal to (>=) 12 weeks.

          -  Able to provide informed consent and be willing to sign an informed consent form.

          -  Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.

          -  Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST])
             =<2.5 x upper limit of normal (ULN) (or =<5 x ULN if in the investigator's opinion the
             elevation is due to extramedullary hematopoiesis).

          -  Serum glutamic pyruvate transaminase (SGPT) alanine aminotransferase (ALT) =<2.5 x ULN
             (or =<5 x ULN if in the investigator's opinion the elevation is due to extramedullary
             hematopoiesis).

          -  Total bilirubin =<3.0 mg/dL (or direct bilirubin < 1 mg/dL).

          -  Creatinine =<3.0 mg/dL.

          -  Absolute neutrophil count >=1000/microliter (mcL).

          -  Platelet count >=50,000/mcL.

          -  Absence of active treatment with systemic anticoagulation and a baseline prothrombin
             time (PT) and activated partial thromboplastin time (aPTT) that does not exceed 1.5 x
             ULN.

          -  Females of childbearing potential must have a negative pregnancy test =<7 days prior
             to registration, unless they are surgically sterile for at least 3 months (i.e.,
             hysterectomy), OR postmenopausal for at least 12 months (follicle-stimulating hormone
             [FSH] >30 U/mL).

          -  Females of childbearing potential must agree to take appropriate precautions to avoid
             pregnancy (with at least 99% certainty) from screening through end of study; permitted
             methods for preventing pregnancy must be communicated to study participants and their
             understanding confirmed.

          -  Males must agree to take appropriate precautions to avoid fathering a child (with at
             least 99% certainty) from screening through follow-up; permitted methods for
             preventing pregnancy should be communicated to the participants and their
             understanding confirmed.

        Exclusion Criteria:

          -  Females who are pregnant or are currently breastfeeding.

          -  Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g.,
             thalidomide), immunosuppressive therapy, corticosteroids > 10 mg/day prednisone or
             equivalent, growth factor treatment (e.g., erythropoietin) or Janus kinase (JAK)
             inhibitor therapy =<14 days prior to registration.

          -  Participants with another active malignancy.

          -  Note: participants with early stage squamous cell carcinoma of the skin, basal cell
             carcinoma of the skin or cervical intraepithelial neoplasia are eligible for
             enrollment.

          -  Known positive status for human immunodeficiency virus (HIV).

          -  Any unresolved toxicity greater or equal to grade 2 from previous anticancer therapy,
             except for stable chronic toxicities not expected to resolve.

          -  Incomplete recovery from any prior surgical procedures or had surgery =<4 weeks prior
             to registration, excluding the placement of vascular access.

          -  Presence of acute active infection requiring antibiotics.

          -  Uncontrolled intercurrent illness or any concurrent condition that, in the
             Investigator's opinion, would jeopardize the safety of the participant or compliance
             with the protocol.