Combining ADI-PEG 20 with venetoclax and azacitidine for treating acute myeloid leukemia
Phase IA/B Combination Study of ADI-PEG 20, Venetoclax and Azacitidine in Patients with Acute Myeloid Leukemia (AML)
This study is testing a new combination of treatments for people with acute myeloid leukemia to see if it can help them better than current options.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 18 Years to 99 Years |
| Sex | All |
| Sponsor | Polaris Group Industry-sponsored |
| Drugs / interventions | chemotherapy |
| Locations | 4 sites (Miami, Florida and 3 other locations) |
| Trial ID | NCT05001828 on ClinicalTrials.gov |
What this trial studies
This phase 1 trial investigates the combination of pegylated arginine deiminase (ADI-PEG 20) with venetoclax and azacitidine in patients with acute myeloid leukemia (AML). The study includes two cohorts: one for patients with relapsed or refractory AML and another for newly diagnosed high-risk AML patients. The trial is open-label and single-arm, focusing on determining the recommended phase 2 dose (RP2D) of ADI-PEG 20 while monitoring for dose-limiting toxicities (DLT). Treatment may continue for up to 24 cycles or until a maximum of 104 doses of ADI-PEG 20 are administered.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with either relapsed/refractory AML or newly diagnosed high-risk AML who are not suitable for intensive chemotherapy.
Not a fit: Patients with low-risk AML or those who are candidates for intensive chemotherapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment combination could improve response rates in patients with high-risk acute myeloid leukemia.
How similar studies have performed: Other studies have shown promising results with similar combinations of venetoclax and azacitidine, suggesting potential for success in this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Miami, Florida and 3 other locations
- Sylvester Comprehensive Cancer Center — Miami, Florida, United States (Recruiting)
- Orchard Healthcare Research Inc — Skokie, Illinois, United States (Withdrawn)
- Levine Cancer Institute — Charlotte, North Carolina, United States (Recruiting)
- MD Anderson Cancer Center — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Nicole DeFord
- Email: ndeford@polarispharma.com
- Phone: 858-452-6688
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.