Experimental combo targets leukemia by starving cancer cells
NCT ID NCT05001828
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a new drug called ADI-PEG 20 added to two standard chemotherapy drugs (venetoclax and azacitidine) for people with acute myeloid leukemia (AML). The study includes 38 adults with either relapsed/refractory AML or newly diagnosed AML who cannot have intensive chemo. The main goal is to find the safest dose and look for early signs that the combination shrinks tumors.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ADI-PEG 20 (a drug that breaks down arginine, an amino acid cancer cells need) combined with venetoclax and azacitidine (standard chemotherapy drugs)
- What this could lead to
- If it works, this could improve treatment options for people with acute myeloid leukemia, especially those who cannot tolerate intensive chemotherapy.
- What could go wrong
- This is a very early, small Phase 1 trial focused on safety and dosing, not proof of effectiveness. The combination may cause side effects or fail to show benefit.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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38 people
The number who actually took part.
- Started
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Apr 2022
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 99 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Cohort 1: Previously treated (relapsed/recurrent) or refractory AML based on the 2022 revision to the World Health Organization (WHO) criteria (Arber 2022) * Cohort 2: Untreated AML per 2022 WHO (Arber 2022) criteria with high-risk features and not a candidate for intensive chemotherapy because of age 60 years or older, age-related comorbidities, cardiac disease, prior anthracycline use, high probability of treatment-related mortality, or otherwise would not benefit from intensive chemotherapy treatment. * Age ≥ 18 years * 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 treatments as follows: 1. Cohort 1: \>2 cycles of prior combination treatment with venetoclax+hypomethelating agent (i.e. azacitidine, decitabine) is exclusionary. All other prior treatment for antecedent hematological disorders and/or for AML is permitted. 2. Cohort 2: Prior treatment for antecedent hematological disorders with venetoclax or chemotherapy or any prior treatment for their AML is exclusionary. However, treatment with other agents, including hydroxyurea or ≤2 cycles of hypomethylating agent (i.e. azacitidine, decitabine), for MDS or myeloproliferative neoplasm is permitted. * Cohort 2: Favorable risk AML per European LeukemiaNet (ELN) 2022 criteria (Döhner 2022) * Known active CNS involvement by leukemia
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Levine Cancer Institute
Charlotte, North Carolina, 28204, United States
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MD Anderson Cancer Center
Houston, Texas, 77030, United States
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Sylvester Comprehensive Cancer Center
Miami, Florida, 33136, United States
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