NCT04797780

Tamibarotene Plus Azacitidine in Participants With Newly Diagnosed RARA-positive Higher-Risk Myelodysplastic Syndrome

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)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 246 (actual)
• Sponsor: Syros Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	Tamibarotene	Administered as specified in the treatment arm
DRUG	Placebo	Administered as specified in the treatment arm
DRUG	Azacitidine	Administered as specified in the treatment arm

Timeline

• Start date: 2021-02-08
• Primary completion: 2024-11-13
• Completion: 2024-11-13
• First posted: 2021-03-15
• Last updated: 2025-04-10
• Results posted: 2025-04-10

Locations

128 sites across 13 countries: United States, Austria, Belgium, Canada, Czechia, France, Germany, Hungary, Israel, Italy, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study