Updates

Texas Children's Cancer and Hematology Center PEPN2312: Imetelstat with Fludarabine and Cytarabine for AML, MDS, or JMML

A Phase 1 Study of GRN163L (Imetelstat) in Combination with Fludarabine and Cytarabine for Patients with Acute Myeloid Leukemia that is in Second or Greater Relapse or that is Refractory to Relapse Therapy; Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia in First or Greater Relapse or is Refractory to Relapse Therapy

Description

Imetelstat is a medication that may inhibit the growth of cancer cells. In this study, doctors will combine imetelstat with the chemotherapy drugs cytarabine and fludarabine to treat acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and juvenile myelomonocytic leukemia (JMML) — cancers that develop in the bone marrow. These conditions are considered high risk when they relapse or do not respond to standard treatments.

The aim of this clinical trial is to determine the safest dose of imetelstat that can be administered alongside fludarabine and cytarabine to halt cancer cell growth. The treatment duration will be 1 - 2 months.

This study will provide valuable insights into the use of imetelstat for treating relapsed or refractory AML, MDS, and JMML. Enrolled patients will have the opportunity to contribute bone marrow and blood samples to improve our understanding of how imetelstat works.

Eligibility Criteria

  • Between 12 months and 18 years old
  • Relapsed or refractory AML, MDS, or JMML
  • Have not received imetelstat

Major Inclusion Criteria

  • Eligible patients must be between the ages of 1 to 18 years at the time of study enrollment with Lansky/Karnofsky performance status of ≥50. 
  • 2nd or greater relapse of AML, 1st or greater relapse of MDS/JMML
  • Refractory disease is allowed
  • Down Syndrome is allowed
  • Therapy related AML is allowed
  • Standard organ function criteria
  • Patients with leukemia must have platelet count >25,000/µL and hemoglobin >8 g/dL (transfusions allowed)

Major Exclusion Criteria

  • Isolated CNS or testicular disease
  • Prior exposure to Imetelstat

For more information, visit clinicaltrials.gov. 

Contact Information

Study Chair
Alexandra Stevens, MD, PhD
Texas Children’s Cancer and Hematology Center
amsteven@texaschildrens.org