QTX-2101 (oral arsenic) plus ATRA for adults with low- or intermediate-risk APL
A Pivotal Open-label Phase 3 Clinical Study Evaluating the Efficacy and Safety of QTX-2101 in Combination With All-trans Retinoic Acid in Newly Diagnosed, Low-risk Acute Promyelocytic Leukemia
This Phase 3 trial tests whether an oral arsenic capsule (QTX-2101) combined with ATRA works well and is safe for adults aged 18–70 with newly diagnosed low- or intermediate-risk acute promyelocytic leukemia.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 150 (estimated) |
| Ages | 18 Years to 71 Years |
| Sex | All |
| Sponsor | Quetzal Therapeutics Industry-sponsored |
| Locations | 4 sites (Duarte, California and 3 other locations) |
| Trial ID | NCT07504458 on ClinicalTrials.gov |
What this trial studies
This open-label Phase 3 study enrolls adults with newly diagnosed low- or intermediate-risk APL confirmed by t(15;17) or PML-RARA. Participants will receive either an oral capsule formulation of arsenic (QTX-2101) plus ATRA or standard IV arsenic trioxide (ATO) plus ATRA according to protocol dosing schedules. The study will track remission rates, safety events, and the pharmacokinetics of the oral formulation with regular clinic visits, laboratory tests, and ECG monitoring. The goal is to determine whether the oral option can match the effectiveness and safety of IV ATO while offering more convenient administration.
Who should consider this trial
Good fit: Adults 18 to under 71 with newly diagnosed, low- or intermediate-risk APL confirmed by t(15;17) or PML-RARA who can comply with study visits, tests, and contraception guidance are ideal candidates.
Not a fit: Patients with high-risk APL, central nervous system leukemia, significant cardiac rhythm problems, uncontrolled infections or severe organ disease, or those who are pregnant or breastfeeding are unlikely to benefit or be eligible.
Why it matters
Potential benefit: If successful, an effective oral arsenic option could reduce or eliminate the need for IV infusions and make treatment more convenient for patients.
How similar studies have performed: ATRA plus IV ATO is an established, highly effective treatment for low-risk APL, while oral arsenic formulations have shown promise in earlier work but have less large-scale phase 3 evidence so far.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Informed Consent 2. Participants must be between 18 and under 71 years of age 3. Participants must have a confirmed diagnosis of APL proven by standard genetic testing (t(15;17) or PML-RARA) 4. Participants must be classified as low- or intermediate-risk APL 5. Participants must be willing and able to comply with the scheduled study visits, treatment plans, laboratory tests, contraception guidance, and other procedures Exclusion Criteria 1. Participants who have significant heart rhythm problems including long QT syndrome, serious arrhythmias, very slow heart rate, or prolonged QTc on ECG 2. Participants who have central nervous system leukemia 3. Participants having serious ongoing medical conditions or infections including uncontrolled infections, severe organ disease, or conditions that make study participation unsafe 4. Participants who are pregnant, breastfeeding, or unwilling to use contraception 5. Participants who are unable to safely take study medication, including severe neuropathy, inability to swallow oral medication, malabsorption issues, or known allergy to ATO or ATRA
Where this trial is running
Duarte, California and 3 other locations
- Quetzal Site 1 — Duarte, California, United States (Recruiting)
- Quetzal Site 4 — Buffalo, New York, United States (Recruiting)
- Quetzal Site 2 — The Bronx, New York, United States (Recruiting)
- Quetzal Site 3 — Charlottesville, Virginia, United States (Recruiting)
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.