Frontline Oral Arsenic Trioxide for APL

• Status: Recruiting
• Trial end date: 2026-06-30
• Target enrollment: 0
• Participant gender: All

Summary

The investigators have formulated an oral preparation of As2O3 (oral-As2O3), and shown that it is efficacious for APL in R1, inducing CR2 in more than 90% of patients [8,9]. Furthermore, in an effort to prevent relapse, the investigators have moved oral-As2O3 forward to the maintenance of CR1. This strategy results in favorable overall-survival (OS) and leukemia-free-survival (LFS) [10], implying that prolonged treatment with oral-As2O3 may prevent relapses.

Current protocols have incorporated i.v.-As2O3 in the treatment of newly-diagnosed APL [11-15]. For regimens comprising As2O3, ATRA and chemotherapy, 5-year OS in excess of 90% is achieved [11-15].

The investigators have also published long-term data showing the use of oral-As2O3 is highly effective and safe in the relapsed and frontline settings [16,17].

In this study, the investigators evaluate the use of oral-As2O3 and ATRA based induction regimens in newly diagnosed patients with APL with no of minimal chemotherapy in a prospective multicentre phase 2 study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: The University of Hong Kong
• Treatments: Arsenic Trioxide

Criteria

Inclusion Criteria:

1. Newly diagnosed APL with t(15;17)(q24;q21) or acute myeloid leukaemia (AML) with variant RARA translocation according to the World Health Organization (WHO) Classification 2016

2. Age ≥18 years

3. Ability and willingness to comply with the study procedures and restrictions

4. Voluntary written informed consent

Exclusion Criteria:

1. ECOG performance score >2

2. Decompensated heart failure with left-ventricular ejection fraction of less than 40% and global hypokinesia on echocardiogram.

3. Prolonged corrected QT interval (QTc) ≥ 500ms, in the absence of electrolyte disturbances and medications known to prolong QTc

4. Significant liver function derangement (Bilirubin > 3 times upper limit normal and/or ALT > 5 times upper limit of normal)

5. Glomerular filtration rate (GRF) by Cockcroft-Gault formula or eGFR (MDRD) of less than 30mL/min

6. Female subject who is lactating or has positive pregnancy test result prior to the first dose of study drug