Phase IA/B Combination Study of ADI-PEG 20, Venetoclax and Azacitidine in Patients With Acute Myeloid Leukemia (AML)

  • End date
    Dec 23, 2025
  • participants needed
  • sponsor
    Polaris Group
Updated on 23 October 2022


Pegylated arginine deiminase (ADI-PEG 20) will be combined with venetoclax and azacitidine for treatment of subjects with previously treated or untreated with high risk factor acute myeloid leukemia (AML). Venetoclax and azacitidine are front-line therapy for such patients, and ADI-PEG 20 will be added to this regimen in a phase IA/B study.


This is an open label, single arm, phase 1 trial with recommended phase 2 dose (RP2D) cohorts based on subject inclusion criteria.

Lead In: 6 patients will be enrolled to be treated with standard dose of azacitidine and venetoclax and the expected RP2D of ADI-PEG 20 (dose level 0). In case of DLT occurring in >1 patient in cycle 1, 6 additional patients will be accrued at dose level -1 of ADI-PEG 20 while keeping the doses of azacitidine and venetoclax unchanged (Dose level -1). Enrollment to cohort 1 and 2 will start after ≤1 patient out of 6 encounters DLT in cycle 1 at one of these dose levels. The 6 patients enrolled at that dose level will be counted for efficacy analysis in Cohort 1. Cohort 1: Relapsed or refractory AML: target response 25%. Historical expectation for venetoclax and azacitidine is 15%.

Cohort 2: Newly diagnosed high risk AML: Target response 55%. Historical expectation for venetoclax and azacitidine is 40%.

Treatment may be continued for a total of 24 cycles, each of 28 days.

Condition Acute Myeloid Leukemia, Adult
Treatment ADI-PEG 20
Clinical Study IdentifierNCT05001828
SponsorPolaris Group
Last Modified on23 October 2022


Yes No Not Sure

Inclusion Criteria

Lead In and Cohort 1
Previously treated AML based on the revised 2017 European LeukemiaNet (ELN) criteria and having ≥10% blasts in bone marrow or peripheral blood
Age ≥18 years
Cohort 2
Untreated AML per ELN criteria with high risk features
Age ≥ 65 years and ineligible for intensive chemotherapy because of older than 75 years, cardiac disease or prior anthracycline use or high probability of treatment-related mortality
Life expectancy reasonably adequate for evaluating the treatment
White blood cell (WBC) count of 10 × 109/L or less. (Use of hydroxyurea to control WBC is allowed till 48 hours prior to protocol treatment)
Adequate renal function: Creatinine ≤ 1.5 x upper limit of normal (ULN) or creatinine clearance > 40 mL/minute (measured or calculated according to the Cockcroft-Gault formula)
Adequate liver function
Total bilirubin ≤ 1.5 x ULN
ALT and AST both ≤ 2.5 x institutional ULN or ≤ 5 times the ULN for patients with leukemic involvement of liver

Exclusion Criteria

Prior treatment for antecedent hematological disorders with hypomethylating agent (more than 2 cycles, those with exposure to <2 cycles will be allowed), venetoclax, or chemotherapy for antecedent hematologic disorders (treatment with other agents including hydroxyurea for myelodysplastic syndrome or myeloproliferative neoplasm is permitted)
Favorable risk AML per ELN 2017 criteria
Known active CNS involvement by leukemia
Clear my responses

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