NCT06778187

A Phase 2 Study of Oral Arsenic Trioxide (Arsenol ®)-Based Low-intensity Treatment for Previously Untreated or Relapsed/Refractory TP53-mutated Myeloid Malignancies

Recruiting now Phase 2 Last updated 5 June 2025

What this trial tests

Phase 2 trial testing Oral arsenic trioxide in Acute Myeloid Leukemia in 30 participants. Currently enrolling.

Timeline

7 February 2025

Primary endpoint
1 February 2027

1 February 2028

Quick facts

Lead sponsor	The University of Hong Kong
Phase	Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	30
Start date	7 February 2025
Primary completion	1 February 2027
Estimated completion	1 February 2028
Sites	1 location across Hong Kong

Drugs / interventions tested

• Oral arsenic trioxide — full drug profile →

Conditions studied

• Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →
• Myelodysplastic Neoplasm — all drugs for Myelodysplastic Neoplasm →
• Chronic Myelomonocytic Leukemia — all drugs for Chronic Myelomonocytic Leukemia →
• TP53 Gene Mutation — all drugs for TP53 Gene Mutation →

Sponsor

The University of Hong Kong

Who can join

18 and older, any sex, with Acute Myeloid Leukemia or Myelodysplastic Neoplasm. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Adverse events
  Time frame: 24 months
  Enumeration and description of adverse events (AEs), serious adverse events (SAEs), and other AEs

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. TP53 -Mutated Myeloid Neoplasms: 2024 Update on Diagnosis, Risk-Stratification, and Management.
   Shah MV, Arber DA, Hiwase DK. · · 2025 · cited 15× · PMID 40066944 · DOI 10.1002/ajh.27655
2. TP53-Mutated Acute Myeloid Leukemia: Unanswered Questions.
   Bruzzese A, Vigna E, Martino EA, Labanca C, et al · · 2025 · cited 4× · PMID 40459096 · DOI 10.1002/hon.70106
3. Discovery of Drugs Targeting Mutant p53 and Progress in Nano-Enabled Therapeutic Strategy for p53-Mutated Cancers.
   Zhang N, Jing Z, Song J, Liang Q, et al · · 2025 · cited 2× · PMID 40563405 · DOI 10.3390/biom15060763
4. Myelodysplastic Neoplasms (MDS): Pathogenesis and Therapeutic Prospects.
   Li X, Zou C, Xiang X, Zhao L, et al · · 2025 · cited 2× · PMID 40563403 · DOI 10.3390/biom15060761
5. Molecular and Clinical Insights into <i>TP53</i>-Mutated MDS and AML.
   Georgantzinos E, Karantanos T. · · 2025 · cited 1× · PMID 41303305 · DOI 10.3390/ijms262210818
6. Crosstalk of thioredoxin system and programmed cell death: from pathophysiology to novel therapy.
   Wu Y, Su Y, Pan F, Jin F, et al · · 2026 · PMID 42155152 · DOI 10.1016/j.redox.2026.104209
7. Mechanistic overview and suggested strategies to overcome BCL-2 inhibitor resistance in <i>TP53-</i>mutated acute myeloid leukemia.
   Iqbal U, Shallis RM. · · 2026 · PMID 41929632 · DOI 10.3389/fcell.2026.1779094
8. TP53-mutated myelodysplastic syndromes and acute myeloid leukemia: a comprehensive overview of targeted approaches.
   Deshpande S, Li WF, Song J, Forsberg M, et al · · 2026 · PMID 41809820 · DOI 10.3389/fonc.2026.1735418