Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of Azacitidine with or without Birinapant for subjects with Higher Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

The Weill Cornell Leukemia Program has recently opened a new clinical trial for men and women who have been diagnosed with Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL) . The study sponsor is TetraLogics Pharmaceuticals and the principal investigator at Weill Cornell is Dr. Ellen Ritchie. For more information about the study, please call Katherine Hassfurter, RN at (212) 746-4882 or e-mail kah9068@med.cornell.edu.

Key Eligibility

  • Men and women age 18 and older
  • Subjects that have not been treated with hypomethylating agents for MDS or CMMoL
  • Histologically confirmed diagnosis of the following conditions:
    – Myelodysplastic Syndrome (MDS)
    – Chronic Myelomonocytic Leukemia (CMMoL)
  • Life expectancy of at least 3 months
  • ECOG score of 0 or 1
  • Detailed eligibility reviewed when you contact the study team

Study Details

This is a randomized, double-blind, clinical trial for men and women with high risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL). The study is evaluating an experimental drug called Birinapant.

Birinapant is being studied as a potential new treatment for MDS or CML. Birinapant removes certain chemicals (proteins) in a cancer cell which leads to the death of cancer cells. From laboratory and animal studies, birinapant is more likely to cause the death of cancer cells than normal cells. Studies combining birinapant with chemotherapy in the human cancer cell laboratory models showed that the addition of birinapant to chemotherapy can result in further blocking of cancer growth, or overcoming cancer resistance to chemotherapy.  In previous research studies, birinapant has shown to be well-tolerated when given alone or in combination with other chemotherapy drugs.

The purpose of this study is to to compare the safety and efficacy (how well it works) of azacitidine plus an investigational drug (birinapant) versus azacitidine plus a placebo (an inactive substance), in patients with higher-risk Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMMoL).