Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)

Active:	Yes
Cancer Type:	Hematopoietic Malignancies Leukemia Unknown Primary	NCT ID:	NCT05183035
Trial Phases:	Phase III	Protocol IDs:	ITCC-101/APAL2020D (primary) NCI-2022-07200 2021-003212-11
Eligibility:	29 Days - 21 Years, Male and Female	Study Type:	Treatment
Study Sponsor:	LLS PedAL Initiative, LLC
NCI Full Details:	http://clinicaltrials.gov/show/NCT05183035

Summary

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone
(fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of
children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who
are unable to receive additional anthracyclines, or in 2nd relapse.

Objectives

Relapse of AML is driven by chemotherapy resistant stem cells. One mechanism of
chemotherapeutic resistance in AML is the overexpression of the protein B-cell lymphoma 2
(BCL-2), an anti-apoptotic protein which sequesters intracellular activators of
apoptosis. Venetoclax is a selective, potent, orally bioavailable, small molecule
inhibitor of B-cell lymphoma (BCL)-2 that restores programmed cell death in cancer cells.

This is a trial for children, adolescents and young adults with 2nd relapsed AML or 1st
relapsed AML unable to receive additional anthracycline.

This is randomized trial of venetoclax in combination with intensive chemotherapy
(fludarabine/cytarabine/gemtuzumab ozogamicin) for the first two cycles that would inform
and evaluate if this agent is an effective option for this population to improve its poor
prognosis. Participants can receive up to two cycles of induction chemotherapy before
hematopoietic stem cell transplantation (HSCT). Participants benefiting from treatment
and who are not able to proceed to HSCT have the possibility to continue to receive
azacitidine in monotherapy (Arm A, control arm) or in combination with venetoclax (Arm B,
experimental arm).

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