Myelodysplastic Syndrome (MDS) Clinical Trial
Protocol: SITE00000035
Full Title
A Randomized, Double-Blind, Phase 3 Study Evaluating the Safety and Efficacy of Venetoclaxin Combination with Azacitidine in Patients Newly Diagnosed with Higher-Risk Myelodysplastic Syndrome (Higher-Risk MDS)
Description
This study enrolls people with Myelodysplastic syndrome (MDS). The purpose of this study is to learn if the study drug, called Venetoclax, can help treat MDS.

People in this study will be randomly selected (like flipping a coin) to be in 1 of two groups. The first group will take venetoclax tablets and an IV infusion or subcutaneous (given under the skin) azacitidine (which is the typical treatment for this condition). Participants in the other group will take placebo tablets and IV or under the skin azacitidine. The study treatment will last about 3-14 months and include around 18 study visits to our office. Compensation for study-related travel may be available. Study takes place at Kaleida Health facilities in Buffalo, NY.

Technical Description

Myelodysplastic syndrome (MDS) represent a heterogeneous group of clonal hematopoietic stem cell disorders with significant morbidity and high mortality (particularly for higher-risk patients). These syndromes are characterized by ineffective hematopoiesis that manifests clinically as cytopenias and a variable rate of transformation to secondary acute myeloid leukemia (sAML).The only curative treatment for higher-risk MDS is an allogeneic stem cell or bone marrow transplantation. However, not all patients are eligible for this intensive treatment approach. If bone marrow transplantation is not possible, patients are typically treated with hypomethylating agents such as azacitidine (AZA).Venetoclax is an oral small molecule inhibitor of BCL-2 which induces apoptosis in preclinical models of multiple hematological malignancies such as chronic lymphatic leukemia (CLL), acute myeloid leukemia (AML), and multiple myeloma (MM), and has shown efficacy in several hematologic malignancies in randomized clinical trials. Venetoclax has demonstrated excellent tolerability through modest efficacy as monotherapy in relapsed/refractory AML. The combinations of venetoclax with AZA have been shown to be effective in AML. In a Phase 1b study, Study M15-531, venetoclax combined with AZA demonstrated favorable safety and efficacy in treatment-nave higherrisk MDS patients.This study will evaluate the efficacy and safety of venetoclax combined with AZA compared to placebo with AZA in patients with newly diagnosed higher-risk myelodysplastic syndrome (MDS). Site For M15-954 (Verona)
Compensation: Yes
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Compensation may include cash, checks, gift cards, debit cards, or incentives like gift baskets, technology items, or merchandise.
Eligibility
Adults ages 18+
Diagnosed with Myelodysplastic syndrome (MDS)
Please contact us for more information about eligibility.
Age Group: Adults
Principal Investigator: Basem Goueli
Contact(s)
Deonna Coleman
dc84@buffalo.edu
Want to Learn More?
Let us know how the study team can reach you. If you do not hear back within 2 business days, reach out to the study team directly at the contact information above or email ctsiresearch@buffalo.edu and someone will assist you.