A Randomized, Double-blind, Placebo-controlled Phase 3 Study of Tamibarotene Plus Azacitidine Versus Placebo Plus Azacitidine in Newly Diagnosed, Adult Patients Selected for RARA-positive Higher-risk Myelodysplastic Syndrome (SELECT MDS-1)
Description
Brief Summary
This study compares the efficacy of Tamibarotene in combination with azacitidine to
azacitidine in combination with placebo in participants who are Retinoic Acid Receptor Alpha
(RARA) positive, and newly diagnosed with higher-risk myelodysplastic syndrome (HR-MDS), and
who have not received treatment for this diagnosis. The primary goal of the study is to
compare the complete remission rate between the two treatment arms.
Detailed Description
A subset of participants have MDS characterized by an overexpression of the RARA gene. A
blood test will be used to identify participants with RARA-positive MDS. Assessment of the
RARA biomarker for study eligibility will be done by collection of blood samples from
potential study participants at the pre-screening visit and testing at a central laboratory.
Participants who meet eligibility requirements will be randomized 2:1 to receive either
Tamibarotene plus azacitidine or placebo plus azacitidine.
Phase
N/AInclusion and Exclusion Criteria
- Participants must be RARA-positive based on the investigational assay.
- Participants must be newly diagnosed with HR-MDS as follows:
- Diagnosis of MDS according to the World Health Organization (WHO) classification and classified by the Revised International Prognostic Scoring System (IPSS R) risk category as very high, high, or intermediate risk.
- Participants must have Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤2. Key
- Participants are suitable for and agree to undergo allogeneic hematopoietic stem cell transplant (HSCT) at the time of screening.
- Participants who need treatment prior to stem cell transplant can receive treatment on this study and stop the study treatment when they are ready to proceed to transplant.
- Participants who received prior treatment for MDS with any hypomethylating agent, lenalidomide, chemotherapy or allogeneic HSCT.
Sites
Please contact the trial administrator to learn more about where you can participate in this trial. Please use the contact form on the right side.