A First-in-Human Phase 1 and Expanded Cohort Study of EPZ-5676 in Advanced Hematologic Malignancies, Including Acute Leukemia With Rearrangement of the MLL Gene

Overview

About this study

The purpose of this study is to determine the safe dose of EPZ-5676, to evaluate the safety of EPZ-5676 in patients with advanced hematologic malignancies, and to conduct a preliminary assessment of the anti-leukemia activity of EPZ-5676 in patients with acute leukemias bearing rearrangements of the MLL gene.

Currently this study is in the MLL-r restricted/expansion phase and is only enrolling patients with rearrangements involving the MLL gene, including 11q23 or partial tandem duplications (PTD).

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Study closed to enrollment

Inclusion Criteria:

  1. Male and female patients aged ≥ 18 years.
  2. Patients with relapsed /refractory AML, ALL, or MLL with rearrangement of the MLL gene, including 11q23 or PTD, are eligible for the expanded cohort:
    • At least one prior therapy;
    • Refractory disease on most recent therapy, or disease recurrence following remission on most recent therapy;
    • Received and failed all known effective therapies for their disease;
    • Not a candidate for allogeneic stem cell transplantation
    • > 10% blasts or biopsy-documented leukemia cutis or myeloid sarcoma.
  3. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
  4. Patients must have the following clinical laboratory values:
    • Serum creatinine ≤2 mg/dL or creatinine clearance > 60 mL/minute;
    • Total bilirubin ≤2.0 times the ULN for the institution, unless considered due to Gilbert's syndrome;
    • ALT or AST ≤ twice the upper limit of normal (ULN), unless considered due to organ leukemic involvement;
    • Absolute neutrophil count ≥1,000/µL (unless due to documented leukemic involvement of the bone marrow at the time of study entry)
    • Platelets ≥100,000/µL (unless due to documented leukemic involvement of the bone marrow at the time of study entry).
    • PT or aPTT < 1.5 times the ULN
  5. Able and willing to give written informed consent.
  6. Life expectancy of at least 3 months

Exclusion Criteria:

  1. Uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements.
  2. Active heart disease
  3. Receiving any other standard treatment for their hematologic malignancy.
  4. Receiving strong CYP3A4 inhibitors/ inducers.
  5. Known history of cerebrovascular accident in the past 6 months.
  6. Known bleeding diathesis.
  7. Known, active (symptomatic) involvement of the central nervous system by leukemia.
  8. On immunosuppressive therapy.
  9. Known active infection.
  10. Pregnant or nursing females.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Raoul Tibes, M.D., Ph.D.

Closed for enrollment

Contact information:

Research Information Center

800-664-4542

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20111976

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