Trial Search Results
Study of Venetoclax in Combination With Chemotherapy in Pediatric Patients With Refractory or Relapsed Acute Myeloid Leukemia or Acute Leukemia of Ambiguous Lineage
The purpose of this study is to test the safety of venetoclax and to find the highest dose of venetoclax that can be given safely when it is combined with one or two chemotherapy drugs (cytarabine or cytarabine and idarubicin). This study tests different doses of venetoclax to see which dose is safer in children with leukemia.
Stanford is currently accepting patients for this trial.
St. Jude Children's Research Hospital
Collaborator: Gateway for Cancer Research
- Drug: Venetoclax
- Drug: Cytarabine
- Drug: Idarubicin
- Drug: Intrathecal Triple Therapy
- Participants must have a diagnosis of AML or acute leukemia of ambiguous lineage
(acute undifferentiated leukemia or mixed phenotype a cute leukemia) and meet the
- Refractory leukemia, defined as persistent leukemia after at least two courses of
induction chemotherapy, OR
- Early relapsed leukemia, defined as the re-appearance of leukemia after the
achievement of remission and within one year of diagnosis, OR
- Relapsed leukemia that is refractory to at least one course of salvage therapy
(i.e., therapy given after the relapse has occurred), OR
- Second or greater relapse
- Patients with late relapses, defined as the re-appearance of leukemia after the
achievement of remission and greater than one year from diagnosis, may be
enrolled in the dose expansion portion of the study only.
- Patients in all categories above must have ≥ 5% blasts in the bone marrow as
assessed by morphology or > 1 blasts in the bone marrow as assessed by flow
cytometry. However, if an adequate bone marrow sample cannot be obtained,
patients may be enrolled if there is unequivocal evidence of leukemia with ≥ 5%
blasts in the peripheral blood. In addition, patients in all categories must not
be eligible to undergo curative therapy, such as immediate SCT, because of
disease burden, time needed to identify a stem cell donor, or other reasons.
- Adequate organ function defined as the following:
- Direct bilirubin ≤ 1.5 x institutional upper limit of normal (ULN)
- AST (SGOT) and ALT (SGPT) ≤ 4 x ULN
- Normal creatinine for age or a calculated creatinine clearance ≥ 60 mL/min/1.73
- Left ventricular ejection fraction ≥ 40% or shortening fraction ≥ 25%
- St. Jude patients must be between 2 years and ≤ 21 years of age, on therapy (active
patient), or within 3 years of completion of therapy. Patients treated at
collaborating sites must be ≤ 24 years old.
- Performance status: Lansky ≥ 50 for patients who are ≤ 16 years old and Karnofsky ≥
50% for patients who are > 16 years old.
- Patients must have fully recovered from the acute effects of all prior therapy and
cannot have evidence of graft-versus-host disease (GVHD)
- Must not be pregnant or breastfeeding. Male or female of reproductive potential must
agree to use effective contraception for the duration of study participation.
- Patients with Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic
leukemia, or bone marrow failure syndromes are not eligible.
- Uncontrolled infection. Infections controlled on concurrent anti-microbial agents are
acceptable, and anti-microbial prophylaxis per institutional guidelines are
- Impairment of GI function or GI disease that may significantly alter the absorption of
Ages Eligible for Study
2 Years - 20 Years
Genders Eligible for Study