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APAL2020D: Venetoclax in Children with Relapsed Acute Myeloid Leukemia (AML)

A Randomized Phase 3 Trial of Fludarabine/Cytarabine/Gemtuzumab Ozogamicin With or Without Venetoclax in Children With Relapsed AML


Leukemia / Lymphoma

Diseases Treated:

Acute Myeloid Leukemia (AML)

Eligibility Overview:

  • Diagnosis of relapsed acute myeloid leukemia (AML)
  • Ages 29 days old to 21 years old
  • Have had AML come back for the first or second time
  • May have heart problems that prevent them from taking anthracyclines
  1. Brief Summary

    Survival rates for children with relapsed AML are poor, particularly for patients who have relapsed a second time. Prior research at St. Jude and elsewhere demonstrated that venetoclax, an oral BCL-2 inhibitor, is a promising agent to potentially improve outcomes for these patients. However, the benefit of venetoclax has not been demonstrated in randomized trials for children with AML or adults with relapsed AML.

    This trial tests in a randomized fashion the addition of venetoclax to a chemotherapy backbone including fludarabine, cytarabine, the gemtuzumab ozogamicin (FLA+GO vs. FLA+GO+venetoclax).

    Primary Objectives

    • Compare the overall survival of venetoclax in combination with fludarabine and high-dose cytarabine (FLA), and gemtuzumab ozogamicin (GO) (FLA+GO+VEN) compared with FLA+GO alone.
    • Assess the duration of response
    • Assess the cumulative incidence of relapse

    Eligibility Criteria

    Inclusion criteria include:

    • At least 29 days old and up to 21 years old
    • AML without FLT3/internal tandem duplication (ITD) mutation in second relapse, who are sufficiently fit to undergo another round of intensive chemotherapy
    • AML without FLT3/internal tandem duplication (ITD) mutation in first relapse who cannot tolerate additional anthracyclines
    • Must have fully recovered from the acute toxic effects of all prior anti-cancer therapy
    • Have adequate organ function

    Exclusion criteria include:

    • Have Down syndrome, acute promyelocytic leukemia or juvenile myelomonocytic leukemia
    • Are currently receiving another investigational drug 
    • Have Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known congenital bone marrow failure syndrome
    • Are pregnant or breastfeeding

    Study Sites

    St. Jude Children’s Research Hospital and other hospitals in North America, Europe, Israel, Australia and New Zealand.

  2. About This Study

    This study will test treatments for acute myeloid leukemia (AML) that has come back (relapsed). The study will test whether adding a new medicine called venetoclax to chemotherapy will improve survival for children with this disease.

    Half of the patients in this study will get venetoclax along with typical chemotherapy. The other half will get typical chemotherapy alone. A computer, rather than your doctor, will choose which treatment you receive.

    Purpose of This Clinical Trial

    To find out if adding venetoclax to standard chemotherapy leads to improved survival for children and young adults with relapsed AML.

    Eligibility Overview

    • At least 29 days old and up to 21 years old
    • Have had AML come back for the first or second time
    • May have heart problems that prevent them from taking anthracyclines
  3. APAL2020D Quick View
    Sponsor Leukemia and Lymphoma Society identifier NCT05183035
    Trial start date June 2022
    Estimated enrollment 98 (up to 10 at St. Jude)
    Study type Interventional
    Study phase Phase 3

    Acute myeloid leukemia (AML)

    Ages 29 days to 21 years
    Principal investigator Seth Karol, MD
    Study site St. Jude Children’s Research Hospital
    For a consultation or to discuss APAL2020D St. Jude Physician/Patient Referral Office


Seth Karol, MD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.