A Phase I Study of EPZ-5676 to Treat Children with Recurrent or Persistent MLL-Mutant Acute Leukemia

Full Title

A Phase 1 Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Patients with Relapsed/Refractory Leukemias Bearing a Rearrangement of the MLL Gene


EPZ-5676 is an investigational drug that blocks a protein involved in leukemia growth called DOT1L. This protein is required for the growth of leukemias that contain an abnormal form of a gene called MLL. EPZ-5676 has shown promise against leukemia in laboratory studies and has also been evaluated in adult patients.

In this study, researchers want to find the highest dose of EPZ-5676 that can be given safely in children with acute leukemia that has come back or continued to grow despite prior treatment. EPZ-5676 is given via a continuous intravenous infusion over 28 days.


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

  • This study is for patients between the ages of 3 months and 18 years.
  • Patients must have acute leukemia with an MLL mutation that has come back after therapy or has stopped responding to therapy.
  • Patients must have recovered from the serious side effects of previous treatments before entering the study.

For more information about this study and to inquire about eligibility, please contact Dr. Neerav Shukla at 212-639-5158.





Disease Status

Relapsed or Refractory