A Phase 1b/2a, Open-label, Non-randomized Study of Birinapant in Combination With 5-azacitidine in Subjects With Myelodysplastic Syndrome Who Are Refractory or Have Relapsed to 5-azacitidine Therapy
This is a Phase 1b/2a, open-label, non-randomized study in male and female subjects with MDS
who are refractory or have relapsed to 5-Azacitidine therapy.
Primary Objective is to determine the maximum tolerated dose (MTD), recommended Phase 2
dose, and pharmacodynamics (PD) of birinapant (TL32711) when administered in combination
with 5-azacitidine (5 AZA) in subjects with myelodysplastic syndrome (MDS) who are
refractory or have relapsed to 5-AZA therapy.
Secondary Objectives are to determine the clinical activity using the International Working
Group (IWG) (Cheson, 2006) Response Criteria for MDS during the Phase 1b dose escalation
stage of the study and in the Phase 2a expansion cohort, to determine the pharmacokinetics
(PK) of birinapant when administered with 5-AZA in plasma and to assess exploratory
translational biomarkers of anti-tumor activity of birinapant in combination therapy.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Maximum tolerated dose (MTD)
United States: Food and Drug Administration
|Miami, Florida 33176|
|Tetralogic research site||Scottsdale, Arizona 85259|
|Tetralogic research site||Buffalo, New York 14263|
|Tetralogic research site||Philadelphia, Pennsylvania 19104|
|Tetralogic research site||Houston, Texas 77030|