Venetoclax for Relapsed Acute Myeloid Leukemia in Children
This study is for children, adolescents, and young adults (ages 29 days to 21 years) with acute myeloid leukemia (AML) that has come back (relapsed). It's looking at whether adding venetoclax to standard chemotherapy (fludarabine, cytarabine, and gemtuzumab ozogamicin) can improve survival. Venetoclax works by targeting a protein called BCL-2, which helps cancer cells survive. Some participants may also receive azacitidine. The study aims to enroll 130 participants and will follow them for up to 5 years to see how long they live after treatment. This trial is for those whose AML has returned a second time, or a first time if they cannot receive more anthracycline chemotherapy.
- Study design
- This is a randomized study, meaning participants are assigned by chance to different treatment groups. It aims to enroll 130 participants.
- What's involved
- Participants will receive up to two cycles of induction chemotherapy. If they benefit and cannot have a stem cell transplant, they may receive maintenance treatment, which could include venetoclax and azacitidine for up to 24 cycles.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 5 years to measure overall survival.
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NCT05183035
Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML)
Recruiting
Phase 3All AgesUpdated 2026-08-26T02:15:34.659160+00:00 on ClinicalTrials.gov
Conditions
Who to contact
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Eligibility criteria
Inclusion
Participants must have enrolled on APAL2020SC, NCT Number: NCT04726241 prior to enrollment on ITCC-101/APAL2020D. (This is only applicable for participants in USA/Canada/Australia/New Zealand sites/Blood Cancer United territory).
Participants must be ≥ 29 days of age and ≤ 21 years of age at enrollment.
Participants must have one of the following:
Untreated second relapse, in participants who are sufficiently fit to undergo another round of intensive chemotherapy, or
Untreated first relapse, in participants who cannot tolerate additional anthracycline containing chemotherapy per investigator discretion.
Participants must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2 (≥ 50% Lansky or Karnofsky score).
Participants must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to start of protocol treatment:
≥ 14 days have elapsed for local palliative RT (small port);
≥ 84 days must have elapsed if prior craniospinal RT or if ≥ 50% radiation of pelvis;
≥ 42 days must have elapsed if other substantial bone marrow (BM) radiation. 7. Stem Cell Infusions (before start of protocol treatment):
≥ 84 days since allogeneic (non-autologous) bone marrow or stem cell transplant (with or without total body irradiation \[TBI\]) or boost infusion (any stem cell product; not including donor lymphocyte infusion \[DLI\]);
No evidence of active graft versus host disease (GVHD). 8. Participants who are receiving cyclosporine, tacrolimus or other agents to treat or prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial. Participants must be off medications to treat or prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant for at least 14 days prior to enrollment. 9. Cellular Therapy: ≥ 42 days after the completion of donor lymphocyte infusion (DLI) or any type of cellular therapy (e.g., modified T cells, natural killer \[NK\] cells, dendritic cells, etc.) before start of protocol treatment. 10. Participants with prior exposure to venetoclax are eligible in this trial.
Adequate organ function:
Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥ 60ml/min/1.73 m\^2, or
Normal serum creatinine based on age/sex 2. Adequate Liver Function defined as:
Direct bilirubin \< 1.5 x upper limit of normal (ULN), and
Alkaline phosphatase ≤ 2.5 x ULN, and
Serum glutamic pyruvic transaminase (SGPT) alanine aminotransferase (ALT) ≤ 2.5 x ULN. If higher transaminases outside these ranges (up to 5x ULN) are due to a radiographically identifiable leukemia infiltrate, the participant will remain eligible. Transaminase elevation up to 5x ULN is also allowed in case of steatosis on echography. 3. Cardiac performance: Minimum cardiac function defined as:
No history of congestive heart failure in need of medical treatment
No pre-treatment diminished left ventricular function on echocardiography (shortening fraction \[SF\] \< 25% or ejection fraction \[EF\] \< 40%)
No signs of congestive heart failure at presentation of relapse.
Participant, parent or guardian must sign and date informed consent and pediatric assent (when required), prior to the initiation of screening or study specific procedures, according to local law and legislation.
Exclusion
Participants who in the opinion of the investigator may not be able to comply with the study requirements of the study, are not eligible.
Participants with Down syndrome.
Participants with Acute promyelocytic leukemia (APL) or Juvenile myelomonocytic leukemia (JMML).
Participants with isolated CNS3 disease or symptomatic CNS3 disease.
Participants with malabsorption syndrome or any other condition that precludes enteral administration of venetoclax.
Participants who are currently receiving an investigational drug other than those specified for this study.
Participants with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known congenital bone marrow failure syndrome.
Participants with known prior allergy to any of the medications used in protocol therapy.
Participants with documented active, uncontrolled infection at the time of study entry.
Known hepatitis C virus (HCV), hepatitis B virus (HBV) (known positive hepatitis B virus (HBV) surface antigen (HBsAg) results), or human immunodeficiency virus (HIV) infection.
Concomitant Medications
Participants who have received strong and moderate CYP3A inducers such as rifampin, carbamazepine, phenytoin, and St. John's wort within 7 days of the start of study treatment.
Participants who have consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or starfruit within 3 days of the start of study treatment.
Participants who have hypersensitivity to the active substance or to any of the excipients listed in summary of product characteristics (SPC).
Pregnancy or Breast-Feeding:
Participants who are pregnant or breast-feeding.
Participants of reproductive potential may not participate unless they have agreed to use a highly effective contraceptive method per Clinical Trial Facilitation Group (CTFG) guidelines for the duration of study therapy and at least 30 days after last dose of venetoclax, or 7 months after gemtuzumab ozogamicin treatment, or for 6 months after the completion of all study therapy, whichever is longer.
Male participants must use a condom during intercourse and agree not to father a child or donate sperm during therapy and for the duration of study therapy and at least 30 days after last dose of venetoclax or 4 months after last dose of gemtuzumab ozogamicin, 6 months from the last dose of cytarabine, or 90-days after last exposure to any other chemotherapy, whichever is longer.
to participants with history of veno-occlusive disease (VOD)/Sinusoidal obstruction syndrome (SOS) grade 3 or 4
to participants with CD33 negative leukemic blasts (determined at local lab)