NCT02147873

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

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

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 118 (actual)
• Sponsor: TetraLogic Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 (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.

Detailed description

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) 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 CMMoL. 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.

Conditions

• Myelodysplastic Syndrome (MDS)
• Chronic Myelomonocytic Leukemia (CMML)

Interventions

Type	Name	Description
DRUG	birinapant
DRUG	Azacitidine
DRUG	Placebo

Timeline

• Start date: 2014-06-01
• Primary completion: 2016-01-01
• Completion: 2016-06-01
• First posted: 2014-05-28
• Last updated: 2016-10-20

Locations

76 sites across 4 countries: United States, Australia, Germany, Spain