Project Title

Study of Azacitidine With or Without Birinapant in Subjects With MDS or CMML.

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

Project Summary

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 (CMML) who are naïve, to azacitidine therapy. Pre-clinical and mechanistic studies support that azacitidine may modulate pathways that enable birinapant-mediated anti-tumor activity.

The primary purpose of this study is to compare the relative effect of azacitidine plus birinapant versus azacitidine plus placebo on response rate in patients with higher-risk MDS, secondary MDS or CMML.

The secondary purpose of this study is to compare effect of azacitidine plus birinapant relative to azacitidine with placebo on:

• Hematologic improvement
• Relapse free survival
• Time to respond
• Change in transfusion requirements
• Duration of response
• Overall survival
• Adverse events

The exploratory objective of this study is to assess exploratory translational biomarkers for antitumor effects.

Blood Disorder

• Myelodysplastic Syndrome
• Chronic Myelomonocytic Leukemia 

Patient Recruitment Details

Patient recruitment status: Closed

Number of Patients (globally)

118 participants.

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