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A Biomarker-Directed Phase 2 Trial of SY-1425, a Selective Retinoic Acid Receptor Alpha Agonist, in Adult Patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

Clinical Trial Details

The purpose of this Phase 2 study is to determine the safety and effectiveness of investigational study drug SY-1425 in treating relapsed/refractory Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) subjects with a certain biomarker in their blood (a blood test that may indicate the drug target is present in your type of AML or MDS). Currently, there is a great unmet need for treating these subject populations. "Investigational" means that SY-1425 has not been approved by the United States Food and Drug Administration (FDA). SY-1425 has been studied in subjects for a similar but different kind of cancer, acute promyelocytic leukemia (APL), and is available to physicians and subjects in Japan, as an approved drug called tamibarotene. 

SY-1425 is a potent and selective retinoic acid receptor alpha (RAR-alpha) agonist. Retinoic acid receptors are transcription factors that are involved in your leukemia.

Key Eligibility: 
  1. Patients must be at least 18 years of age.
  2. Patients must have A. relapsed and/or refractory non-APL AML that has failed to achieve a complete remission (CR) or partial remission (PR) following standard induction therapy, or has relapsed after any duration of CR or PR who are positive for the RARA super-enhancer associated biomarker OR B. Relapsed and/or refractory high-risk MDS OR C. Newly diagnosed, treatment-naive non-APL AML in patients greater than or equal to 60 years of age.
  3. Patients must be positive for the RARA super-enhancer associated biomarker or IRF8 biomarker. 

   Detailed eligibility will be reviewed when you contact the study team.

Study contact by location

Upper East Side - Manhattan


Yulia Dault, RN
(212) 746-4829

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:






Open to Enrollment

Age Group