Phase 1b Acute Myelogenous Leukemia (AML) Study With ABT-199 + Decitabine or Azacitidine (Chemo Combo)


About this study

This is a Phase 1b, open-label, non-randomized, multicenter study to evaluate the safety of orally administered ABT-199 combined with decitabine or azacitidine and the preliminary efficacy of one of these combinations. In addition, there is a DDI sub-study only at a single site, to assess the pharmacokinetics and safety of ABT-199 in combination with posaconazole.

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.

Inclusion Criteria:

  1. Subjects must have histological confirmation of AML by WHO criteria and be ineligible for treatment with a standard cytarabine and anthracycline induction regimen due to co-morbidity or other factors.
  2. Subjects must have received no prior treatment for AML with the exception of hydroxyurea, allowed through the first cycle of study treatment. NOTE: Subject may have been treated for prior Myelodysplastic Syndrome (excluding hypomethylating agents).
  3. Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance status of 0 to 2.
  4. Subject must have adequate renal function as demonstrated by a calculated creatinine clearance ≥ 50 mL/min; determined via urine collection for 24-hour creatinine clearance or by the Cockcroft Gault formula.
  5. Subject must have adequate liver function.

Exclusion Criteria:

  1. Subject has received treatment with a hypomethylating agent and/or cytarabine for a pre-existing myeloid disorder.
  2. Subject must not have favorable risk cytogenetics as categorized by the NCCN Guidelines Version 2, 2014 for Acute Myeloid Leukemia.
  3. Subject has t(8;21), inv(16) or t(15;17) karyotype abnormalities.
  4. Subject has acute promyelocytic leukemia (French-American-British Class M3 AML).
  5. Subject has known active CNS involvement with AML.
  6. Subject has received the following within 7 days prior to the initiation of study treatment:
    1. Potent CYP3A inducers such as rifampin, carbamazepine, phenytoin, and St. John's wort;
    2. Warfarin or requires the use of warfarin and its equivalent drugs, such as phenprocoumon; (due to potential drug-drug interactions that may potentially increase the exposure of warfarin and complications of anticoagulant effect).
    3. CYP3A inhibitors such as fluconazole, ketoconazole, and clarithromycin.
  7. Subject has a history of other malignancies prior to study entry, with the exception of:
    1. Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast;
    2. Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin;
    3. Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent.
  8. Subject has a white blood cell count > 25 × 10^9/L. Note: Hydroxyurea is permitted to meet this criterion.
  9. Subject is a candidate for a bone marrow or stem cell transplant within 12 weeks after study enrollment.

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:

Cancer Center Clinical Trials Referral Office


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