Oral arsenic trioxide plus vitamin C with low-intensity therapy for TP53-mutated AML/MDS/CMML

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label Phase 2 study plans to enroll about 30 adults with TP53-mutated acute myeloid leukemia, myelodysplastic neoplasm, or chronic myelomonocytic leukemia, split roughly between treatment-naïve and relapsed/refractory cohorts. Participants will receive oral arsenic trioxide (Arsenol®) together with ascorbic acid and an investigator-selected low-intensity therapy—typically a hypomethylating agent given with or without venetoclax—on 28-day cycles. The study will monitor tolerability, hematologic responses, and molecular responses as primary signals of activity and safety. Pending tolerability and signal of benefit, investigators may plan a future randomized Phase 2 efficacy study.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Willing and able to provide informed consent
2. Age ≥18 years
3. Diagnosis of acute myeloid leukemia (AML), myelodysplastic neoplasm (MDS) or chronic myelonocytic leukaemia (CMML) by World Health Organization (WHO) 2022 criteria (1, 3)
4. Presence of TP53 mutation
5. Previously untreated patients for Cohort A (Treatment-naïve), or Patients failing 1 or more lines of prior treatment for Cohort B (Relapsed and Refractory)
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
7. Women of childbearing potential and fertile men must agree to use an approved method of contraception from Screening until 30 days after the last dose of oral arsenic trioxide, ascorbic acid, venetoclax and azacitidine/decitabine/oral-decitabine-cedazuridine.

Exclusion Criteria:

Inclusion Criteria

1. Willing and able to provide informed consent
2. Age ≥18 years
3. Diagnosis of acute myeloid leukemia (AML), myelodysplastic neoplasm (MDS) or chronic myelonocytic leukaemia (CMML) by World Health Organization (WHO) 2022 criteria (1, 3)
4. Presence of TP53 mutation
5. Previously untreated patients for Cohort A (Treatment-naïve), or Patients failing 1 or more lines of prior treatment for Cohort B (Relapsed and Refractory)
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
7. Women of childbearing potential and fertile men must agree to use an approved method of contraception from Screening until 30 days after the last dose of oral arsenic trioxide, ascorbic acid, venetoclax and azacitidine/decitabine/oral-decitabine-cedazuridine.

Exclusion Criteria

1. Use of an investigational agent within 14 days of study treatment (or at least 7 half-lives of that agent, whichever is longer), prior to the first dose of oral arsenic trioxide
2. Known hypersensitivity to arsenic trioxide, ascorbic acid, venetoclax or azacitidine/decitabine/oral-decitabine-cedazuridine or their excipients.
3. Uncontrolled, active infection
4. Major surgery within 4 weeks of starting the study drug, or not recovered from side effects of surgery
5. Any other serious medical conditions that could compromise study participation, in the opinion of the investigator
6. Known HIV infection or known, active hepatitis B or hepatitis C infection
7. Concurrent second active and non-stable malignancy (patients with a concurrent second active but stable malignancy, i.e., non-melanoma skin cancers, are eligible)
8. Known history of long QT syndrome (LQTS) or corrected QT interval by Fridericia formula (QTcF) ≥ 480 ms
9. Evidence at the time of Screening of significant renal or hepatic insufficiency (unless due to hemolysis) as defined by any of the following local laboratory parameters:

   1. Calculated glomerular filtration rate (GFR; using the Cockcroft-Gault equation or eGFR; using CKD-EPI) \< 40 mL/min
   2. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3 x the local upper limit of normal
10. Pregnant or lactating females, or females planning to become pregnant at any time during the study
11. Unwilling or unable to comply with the study protocol

Where it's running

• Queen Mary Hospital, Hong Kong
  Hong Kong, Hong Kong Recruiting

Study contacts

• Principal investigator: Harinder Gill, MD — The University of Hong Kong
• Study coordinator: Harinder Gill, MD
• Email: gillhsh@hku.hk
• Phone: +852 2255 5859

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.