Clinical Trial 23059

• Overview

  Study 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

  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.

  Objective:

  Primary: Characterize and compare the CR rate of tamibarotene plus azacitidine vs. placebo plus azacitidine. Secondary: Characterize and compare the TI rate of tamibarotene plus azacitidine vs. placebo plus azacitidine. Characterize and compare the ORR of tamibarotene plus azacitidine vs. placebo plus azacitidine. Characterize the DOCR and duration of overall response of tamibarotene plus azacitidine or placebo plus azacitidine. Characterize the time to CR and time to initial response of tamibarotene plus azacitidine vs. placebo plus azacitidine. Characterize and compare the EFS of tamibarotene plus azacitidine vs. placebo plus azacitidine. Compare changes in HRQOL of tamibarotene plus azacitidine vs. placebo plus azacitidine. Characterize the safety of tamibarotene plus azacitidine vs. placebo plus azacitidine.

• Treatments

  Therapies:

  Chemotherapy (NOS); Therapy (NOS)

  Medications:

  Tamibarotene/Placebo (); azacitidine (5-azacitidine)

• Inclusion Criteria

  > Inclusion 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 or less

• Exclusion Criteria

  > Exclusion Criteria:
  • 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.

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