A Phase 3 Study of Standard Chemotherapy with or without Venetoclax in Children and Adolescents with Relapsed Acute Myeloid Leukemia


Full Title

Randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML [ITCC-101/APAL2020D]


Researchers in this study want to determine if combining the drug venetoclax with standard chemotherapy may be more effective than standard chemotherapy alone in children and adolescents with relapsed acute myeloid leukemia (AML). Venetoclax blocks Bcl-2, a protein that helps cancer cells to survive and resist the effects of cancer treatments. By blocking Bcl-2, venetoclax may kill cancer cells and/or make other treatments more effective.

Participants in this study will be randomly assigned to receive standard chemotherapy (fludarabine, cytarabine, and gemtuzumab ozogamicin) alone or in combination with venetoclax. The chemotherapy drugs are given intravenously (by vein) and venetoclax is taken orally (by mouth).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have AML that came back despite prior treatment and does not contain a FLT3/ITD mutation.
  • The serious side effects of previous treatments must go away before the study therapy is given.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for people older than 28 days and under age 22 years.


For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.




Phase III (phase 3)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID