Study of Azacitidine With or Without Birinapant in Subjects With MDS or CMMoL

Overview

  • Study type

    Interventional
  • Study phase

    II
  • Study IDs

  • 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.
  • 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.

  • Site IRB
    • Scottsdale/Phoenix, Arizona: 14-004541
    • Jacksonville, Florida: 14-004541
    NCT ID: NCT02147873
    Sponsor Protocol Number: TL32711-0094

About this study

This is a randomized double blind placebo controlled study of azacitidine with or without birinapant in subjects with higher risk Myelodysplastic syndrome, secondary MDS or myelomonocytic leukemia (CMMoL) who are naïve, to azacitidine therapy. Pre-clinical and mechanistic studies support that azacitidine may modulate pathways that enable birinapant-mediated anti-tumor activity.

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:

  • Morphologically confirmed diagnosis of MDS/CMMoL according to FAB or WHO classification, including RAEB-t and MDS/MPN
  • International prognostic score-revised (IPSS-R) of >3.5 (Intermediate, High or Very High)
  • Previously untreated with hypomethylating agents for MDS/CMMoL
  • Performance status of 0, 1 or 2 by the ECOG scale
  • Adequate renal and liver function
  • Female subjects of childbearing potential must have a negative serum pregnancy test at screening within 96 hours prior to the first study dose.
  • Female subjects of childbearing potential and male subjects with partners of childbearing potential should ensure use of a highly effective method of birth control as defined by the investigator, for example, those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly during the study and for a period of 3 months following the last dose of any drug administered during the study.

Exclusion Criteria:

  • Relapsed or refractory to hypomethylating agents
  • Acute myeloid leukemia (AML), except those patients with RAEB-t who are not candidates for intensive AML therapy.
  • Participated in any interventional study within 4 weeks of randomization or 5 half lives (whichever is longer).
  • Received any hematopoietic growth factors within 14 days prior to screening.
  • Prior malignancy or secondary malignancy within the prior 2 years (except in situ cervical cancer, squamous cell carcinoma or basal cell carcinoma of the skin).
  • known diagnosis of human immunodeficiency virus or chronic active Hep B or C.
  • Uncontrolled hypertension
  • Impaired cardiac function, uncontrolled cardiac arrhythmias despite medications, or clinically significant cardiac disease
  • Lack of recovery of prior adverse events to Grade ≤1 severity (National Cancer Institute Common Terminology Criteria for Adverse Events version 4) (except alopecia) due to therapy administered prior to the initiation of study drug dosing.
  • Nursing or pregnant.
  • Known allergy or hypersensitivity to any of the formulation components
  • Any concurrent disease and/or medical condition that, in the opinion of the Investigator, would prevent the subject's participation.
  • History of cranial nerve palsy.
  • Being treated with anti-TNF therapies or has been treated with an anti-TNF therapy within 5 half-lives of randomization.

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Scottsdale/Phoenix, Ariz.

Mayo Clinic principal investigator

Jeanne Palmer, M.D.

Closed for enrollment

Contact information:

Research Information Center

800-664-4542

Jacksonville, Fla.

Mayo Clinic principal investigator

Jeanne Palmer, M.D.

Closed for enrollment

Contact information:

Research Information Center

800-664-4542

.
CLS-20145332

Mayo Clinic Footer