Clinical trial · Interventional
Oral-ATO for TP53-mutated Myeloid Malignancies
A Phase 2 Study of Oral Arsenic Trioxide (Arsenol ®)-Based Low-intensity Treatment for Previously Untreated or Relapsed/Refractory TP53-mutated Myeloid Malignancies
NCT06778187CI-TRIAL-00090809recruitingPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This is an open-label, phase 2 study of oral arsenic trioxide (Arsenol ®) in combination with ascorbic acid and investigator choice of low-intensity therapy in patients with previously untreated or relapse/refractory TP53-mutated acute myeloid leukemia (AML), myelodysplastic neoplasm (MDS), chronic myelomonocytic leukemia (CMML).
Conditions
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
| Arsenic Trioxide | — | UNRESOLVED | — |
| Chronic Myelomonocytic Leukemia | Chronic Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Myelodysplastic Neoplasm | Myelodysplastic Syndrome | ALIAS | 0.90 |
| TP53 Gene Mutation | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Oral arsenic trioxide | Drug | Arsenic Trioxide | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Oral arsenic trixoide
- description
- Patients will be treated in 28-day cycles. Each cycles will comprise: Oral ATO (10mg/day or 0.15mg/kg/day in patients \< 50kg) from Days 1-14 PLUS: * Oral ascorbic acid (1000mg/day) from Days 1-14 * Azacitidine (75mg/m2/day s.c. or i.v.) from days 1-7; OR Decitabine (20mg/m2/day i.v.) from days 1-5; OR Oral-decitabine-cedazuridine (1 tablet/day) from days 1-5. * Venetoclax (100mg-400mg/day) from Days 1-14 (if used)
- interventionNames
- Drug: Oral arsenic trioxide
Primary outcomes (1)
- measure
- Adverse events
- timeFrame
- 24 months
- description
- Enumeration and description of adverse events (AEs), serious adverse events (SAEs), and other AEs
Secondary outcomes (2)
- measure
- Response rates
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
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
References
Publications (1)
- BACKGROUNDSallman DA, Stahl M. TP53-mutated acute myeloid leukemia: how can we improve outcomes? Blood. 2025 Jun 12;145(24):2828-2833. doi: 10.1182/blood.2024024245. PMID 39236289