A Phase II Study of SY-1425 (Tamibarotene) in Patients with Recurrent or Persistent Acute Myeloid Leukemia or Myelodysplastic Syndromes

Full Title

SY-1425-201: 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)

Purpose

SY-1425 (tamibarotene) is an investigational drug designed to inhibit cancer growth by targeting proteins on cancer cells called RARA and IRF8. SY-1425 is a form of retinoic acid (a chemical cousin of vitamin A).

In this study, researchers are assessing the safety and effectiveness of SY-1425 in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) that has come back or continues to grow despite prior therapy. Older patients with AML who have not yet been treated and who are not candidates for standard AML therapies may also participate, as well as patients with low-risk MDS who need blood transfusions and who cannot receive erythropoietin. SY-1425 is taken orally (by mouth).

Eligibility

To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have AML or MDS that has come back or continues to grow despite treatment.
  • Older patients with AML (age 60 and older) who have not yet been treated and who are not candidates for standard AML therapies may also participate, as well as patients with low-risk MDS who need blood transfusions and who cannot receive erythropoietin.
  • Patients’ cancers must contain RARA or IRF8.
  • At least 2 weeks must pass between the completion of prior therapies and entry into the study.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Eytan Stein at 212-639-3314.

Protocol

17-031

Phase

II

Investigator

Co-Investigators