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

a study on Myelodysplastic Syndrome

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Irvine, California and other locations
Dates
study started
completion around
Principal Investigator
by Deepa Jeyakumar

Description

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.

Official Title

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)

Details

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.

Keywords

Myelodysplastic Syndromes, Higher-Risk, Retinoic Acid Receptor Alpha (RARA) positive, Newly Diagnosed, Preleukemia, Syndrome, Azacitidine, Tamibarotene, Tamibarotene + Azacitidine

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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.

You CAN'T join if...

  • 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.

Locations

  • UC Irvine accepting new patients
    Irvine California 92697 United States
  • University of California, Los Angelas accepting new patients
    Los Angeles California 90095 United States

Lead Scientist at UC Irvine

  • Deepa Jeyakumar
    Associate Clinical Professor, Medicine, School of Medicine. Authored (or co-authored) 29 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Syros Pharmaceuticals
ID
NCT04797780
Phase
Phase 3 Myelodysplastic Syndrome Research Study
Study Type
Interventional
Participants
Expecting 550 study participants
Last Updated
Contact us about this trial