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Study of Azacitidine With or Without Birinapant in Subjects With MDS or CMMoL
Trial ID: NCT02147873
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.
A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of Azacitidine With or Without Birinapant With a Single Arm Open-Label Run-In Phase in Subjects With Higher Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia
key 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
- 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
Key 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.
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