Study of MLN4924 Plus Azacitidine in Treatment-Naïve Patients With Acute Myelogenous Leukemia (AML) Who Are 60 Years or Older

  • Study type:

    Interventional What is this?

    Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • Study phase:

    I What is this?

    During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

Study IDs

  • Site IRB:

    • Jacksonville, Florida: 13-007425
  • NCT ID:

    NCT01814826
  • Sponsor Protocol Number:

    C15009

About this study

This study is an open-label, multicenter, phase 1b, dose-escalation study of MLN4924 in combination with azacitidine in adult patients with AML. The patient population will consist of patients 60 years of age or older, previously diagnosed with World Health Organization (WHO)-defined AML, who are unlikely to benefit from standard induction therapy and who have not received definitive treatment for AML or prior treatment with azacitidine or decitabine.

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. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria:

  • Patients with WHO-defined AML, 60 years of age or older, who are unlikely to benefit from standard induction therapy, defined as having at least 1 of the following:
    1. Greater than or equal to 75 years of age
    2. Antecedent hematologic disease
    3. Known adverse cytogenetic risk
    4. Eastern Eastern Cooperative Oncology Group (ECOG) PS = 2
  • Patient must not have received definitive treatment for AML, defined as any prior chemotherapy with antileukemic activity
  • ECOG PS 0 to 2
  • Expected survival longer than 3 months from enrollment in the study
  • Female patients who are post menopausal, surgically sterile, or agree to practice 2 effective methods of contraception or agree to practice true abstinence
  • Male patients who agree to practice effective barrier contraception or agree to practice true abstinence
  • Voluntary written consent must be given before performance of any study-related procedure
  • Suitable venous access for the study-required blood sampling
  • Adequate clinical laboratory values during the screening period as specified in the protocol
  • Able to undergo bone marrow aspiration and biopsy at screening

Exclusion Criteria:

  • Previous treatment with azacitidine or decitabine
  • Known favorable cytogenetic risk
  • Any serious medical or psychiatric illness
  • Treatment with any investigational products
  • Known hypersensitivity to azacitidine or mannitol
  • Acute promyelocytic leukemia as diagnosed by morphologic examination of bone marrow, by fluorescent in situ hybridization or cytogenetics of peripheral blood or bone marrow, or by other accepted analysis
  • Active uncontrolled infection or severe infectious disease
  • Major surgery within 14 days before the first dose of study drug
  • Life-threatening illness unrelated to cancer
  • Clinically uncontrolled central nervous system (CNS) involvement
  • Known human immunodeficiency virus (HIV) positive
  • Known hepatitis B surface antigen-positive, or known or suspected active hepatitis C infection Known hepatic cirrhosis or severe pre-existing hepatic impairment Known cardiac/cardiopulmonary disease
  • Left ventricular ejection fraction
  • Known moderate to severe chronic obstructive pulmonary disease, interstitial lung disease, and pulmonary fibrosis
  • Body mass index > 40 kg/m²
  • Treatment with CYP3A inducers within 14 days before the first dose of MLN4924
  • Systemic antineoplastic therapy or radiotherapy within 14 days before the first dose of study drug, except for hydroxyurea
  • Patients who are unwilling or unable to refrain from using hydroxymethylglutaryl coenzyme A (HMG CoA) reductase inhibitors (statins) starting 5 days before the initial study drug administration and throughout the study will not be permitted to enroll

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

James Foran, M.D.

Contact us for the latest status

Cancer Center Clinical Trials Referral Office

855-776-0015