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Venetoclax added to fludarabine, cytarabine and gemtuzumab ozogamicin (drug combination) in children with relapsed acute myeloid leukemia

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study looks at children whose acute myeloid leukemia has come back after treatment (called relapsed). The medicines being tested are a pill called Venetoclax taken by mouth, and three other medicines given by injection: fludarabine, cytarabine, and gemtuzumab ozogamicin. These drugs are used together to try to kill leukemia cells.

The goal of the trial is to find out whether adding Venetoclax to the standard three‑drug mix can improve overall survival, which means living longer after the treatment starts.

In this phase 3 study, children are placed by chance into one of two groups: one group receives the three standard drugs, and the other group receives the same three drugs plus the oral Venetoclax. Treatment is given in cycles over several weeks, and the children are checked regularly to see how they are doing and to record any side effects. The study follows each participant for a period of time to see how long they live and how they feel.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and consent

    After joining the trial you sign an informed consent form that explains the purpose of the study and the procedures that will be performed.

  2. Step 2

    Baseline assessments

    Initial medical examinations, blood tests, and imaging are performed to document the current status of acute myeloid leukemia before treatment begins.

  3. Step 3

    Chemotherapy administration (fla+go)

    You receive fludarabine at a dose of 75 mg/m2 given by subcutaneous injection.

    You receive cytarabine at a dose of 2 gm/m2 given by intravenous infusion.

    You receive gemtuzumab ozogamicin at a dose of 3 mg/m2 given by intravenous infusion.

    These three medicines are administered according to the schedule defined in the trial protocol during each treatment cycle.

  4. Step 4

    Oral venetoclax administration (combination arm only)

    If you are assigned to the combination arm you take venetoclax 600 mg by mouth each day as an oral suspension or film‑coated tablet, depending on the formulation supplied.

    The daily dose is continued for the length of each treatment cycle as specified by the study protocol.

  5. Step 5

    Monitoring and follow‑up

    Throughout the treatment period you undergo regular blood tests, physical examinations, and other assessments to evaluate response to therapy and to check for side effects.

    Any required adjustments to medication doses are made based on the results of these evaluations.

  6. Step 6

    Completion of treatment and final evaluation

    After the planned number of treatment cycles you complete the study medication regimen.

    A final set of assessments is performed to determine overall survival and disease status at the end of the trial.

Who can join the trial?

16 criteria

  • Must be a child, teenager, or young adult with acute myeloid leukemia (AML) that does NOT have the FLT3/ITD mutation; the test for this mutation should be done at the time of the current relapse, but an earlier negative test is acceptable.
  • The leukemia must be either an untreated second relapse in a patient who is healthy enough for another round of intensive chemotherapy, or an untreated first relapse in a patient who cannot tolerate more chemotherapy that contains a drug called anthracycline.
  • Must not be taking any medicines to treat or prevent graft‑versus‑host disease (a complication after a bone‑marrow transplant) or organ‑rejection drugs for at least 14 days.
  • Must be at least 42 days after receiving any type of cellular therapy such as a stem‑cell transplant.
  • Must have adequate organ function:
    • Kidney: estimated glomerular filtration rate (eGFR) ≥ 60 ml/min/1.73 m² or a normal serum creatinine for age/sex.
    • Liver: total or direct bilirubin ≤ 1.5 times the upper limit of normal (ULN), alkaline phosphatase ≤ 2.5 × ULN, and ALT (a liver enzyme) ≤ 2.5 × ULN. Higher levels are allowed if they are caused by leukemia in the liver or fatty liver seen on imaging.
    • Heart: no history of congestive heart failure that needs medication, normal left‑ventricular function on an echocardiogram (ejection fraction ≥ 40% or shortening fraction ≥ 25%), and no signs of heart failure at the time of relapse.
    • Must sign an informed consent form (and assent form for children, when required) before any screening or study procedures begin.
    • Must have a performance status score of 0, 1, or 2 on the ECOG scale (or a Lansky/Karnofsky score of at least 50%), indicating they are able to carry out most daily activities.
    • Must have fully recovered from any immediate side effects of previous cancer treatments and must wait the required time after those treatments before joining the study (exact timing is detailed in the study protocol).
    • Must not have received any strong chemotherapy drugs (cytotoxic chemotherapy) within the past 14 days, except that low‑dose steroids, low‑dose cytarabine, or hydroxyurea may be given up to 24 hours before starting the study treatment.
    • Must wait at least 21 days after the last dose of any antibody‑drug conjugate (a special type of cancer‑targeting drug).
    • Must wait at least 21 days after finishing any interleukins, interferons, or cytokines (types of immune‑system medicines).
    • Must wait at least 14 days after the last dose of a long‑acting blood‑cell growth factor, or at least 7 days after a short‑acting growth factor.
    • If they had radiation therapy, it must have been completed between 14 and 84 days ago, depending on how large the treated area was.
    • Must be at least 84 days since receiving an allogeneic bone‑marrow or stem‑cell transplant (or a boost infusion) and must have no active graft‑versus‑host disease.

Who cannot join the trial?

13 criteria

  • Patients who, in the doctor's view, may not be able to follow the study rules and visits.
  • People who have a known infection with hepatitis C virus (HCV), hepatitis B virus (HBV) (a positive HBV surface antigen test), or human immunodeficiency virus (HIV).
  • Patients who have taken strong or moderate CYP3A enzyme inducers—such as the antibiotics rifampin, the seizure medicines carbamazepine or phenytoin, or the herbal product St. John’s wort—within 7 days before starting the trial, or who have eaten grapefruit, Seville oranges (including marmalade) or starfruit within 3 days, or who are allergic to the study drug or any of its inactive ingredients.
  • Women who are pregnant or breastfeeding, and anyone who cannot use a highly effective birth‑control method as required for the study period and after the last dose of study medicines.
  • Patients who have had a severe form of veno‑occlusive disease (VOD) or sinusoidal obstruction syndrome (SOS), or whose leukemia cells do not have the marker CD33 (these patients cannot receive the drug gemtuzumab ozogamicin).
  • People with Down syndrome.
  • Patients diagnosed with acute promyelocytic leukemia (APL) or juvenile myelomonocytic leukemia (JMML).
  • Patients who have disease in the central nervous system classified as CNS‑3 (either isolated or causing symptoms).
  • Individuals with a malabsorption disorder or any condition that makes it impossible to take the study drug venetoclax by mouth.
  • Patients who are currently receiving another investigational (still‑testing) drug that is not part of this study.
  • People with known inherited bone‑marrow failure conditions such as Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or any similar congenital disorder.
  • Patients who have a known allergy to any of the medicines used in the trial.
  • Patients who have an active, uncontrolled infection at the time they would join the study.
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Investigated drugs

  • Venetoclax

    is an oral medicine that blocks a protein cancer cells use to avoid death. In this study it is added to standard chemotherapy to see if it helps children with relapsed AML live longer.

  • Fludarabine

    is a chemotherapy drug given by injection under the skin. It interferes with the DNA inside leukemia cells, making it harder for the cancer to grow.

  • Cytarabine

    is a chemotherapy medication given through a vein. It targets rapidly dividing blood‑cancer cells and is a key part of the treatment for AML.

  • Gemtuzumab ozogamicin

    is a targeted therapy delivered by IV. It attaches to a specific marker on leukemia cells and carries a toxin that kills those cells while sparing most normal cells.

  • Azacitidine

    is a drug that changes how genes are turned on or off in blood‑cancer cells. It is given by injection under the skin and works as a type of chemotherapy for certain blood cancers.

  • FLA+GO regimen

    refers to the combination of fludarabine, high‑dose cytarabine, and gemtuzumab ozogamicin. This standard chemotherapy mix is used to treat children whose AML has come back, and the trial tests whether adding venetoclax improves outcomes.

What is already known about the treatment

Venetoclax - It is taken by mouth as a suspension or film‑coated tablet. It is an approved drug for certain blood cancers, including acute myeloid leukemia, and is widely studied in medical literature. It works by binding to the BCL‑2 protein, which helps cancer cells survive, and this binding triggers the cells to die. It belongs to the class of selective BCL‑2 inhibitors, a type of targeted therapy. Azacitidine - It is given by subcutaneous injection. It is an approved treatment for myelodysplastic syndromes and for some patients with acute myeloid leukemia, and many clinical studies have examined its use. It is a nucleoside analog that gets incorporated into DNA and blocks abnormal DNA methylation, leading to death of abnormal blood cells. It is classified as a hypomethylating antimetabolite. Cytarabine - It is administered by intravenous infusion. It is a standard, approved chemotherapy for acute myeloid leukemia and is often used in high doses in clinical trials. It mimics the building block of DNA and stops the cancer cells from making new DNA, which kills them. It belongs to the antimetabolite class of antineoplastic agents. Fludarabine phosphate - It is given by intravenous infusion. It is an approved drug for chronic lymphocytic leukemia and is also used in some AML regimens, with extensive research supporting its use. It is a purine analog that interferes with DNA synthesis, causing cancer cells to die. It is classified as a purine‑type antimetabolite. Gemtuzumab ozogamicin - It is given by intravenous infusion. It is an approved, orphan‑designated therapy for acute myeloid leukemia and is studied in many trials. It combines an antibody that binds the CD33 protein on AML cells with a toxin called calicheamicin; the antibody delivers the toxin directly to the cancer cells, leading to their death. It is classified as an antibody‑drug conjugate, a form of targeted therapy.

Investigated diseases

Acute Myeloid Leukemia - Acute Myeloid Leukemia is a cancer that begins in the bone marrow, where blood cells are made. It causes the rapid growth of abnormal white blood cells that do not function properly. These abnormal cells crowd out normal blood cells, leading to tiredness, easy bruising, and increased risk of infection. The disease can spread quickly within the marrow and may return after an initial response.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-510160-12-00Protocol codeITCC-101/APAL2020DEstimated enrolment130 patientsSponsorPrincess Maxima Center For Pediatric Oncology

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