RecruitingPhase 3NCT07504458

Pivotal Open-label Phase 3 Clinical Study of QTX-2101 in Adult Patients With Acute Promyelocytic Leukemia

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


Sponsor

Quetzal Therapeutics

Enrollment

150 participants

Start Date

Apr 1, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

This Phase 3 study in adult participants with newly diagnosed low-risk APL will evaluate the efficacy, safety, and PK of an oral capsule formulation of ATO, in combination with ATRA.


Eligibility

Min Age: 18 YearsMax Age: 71 Years

Inclusion Criteria5

  • Informed Consent
  • Participants must be between 18 and under 71 years of age
  • Participants must have a confirmed diagnosis of APL proven by standard genetic testing (t(15;17) or PML-RARA)
  • Participants must be classified as low- or intermediate-risk APL
  • Participants must be willing and able to comply with the scheduled study visits, treatment plans, laboratory tests, contraception guidance, and other procedures

Exclusion Criteria5

  • Participants who have significant heart rhythm problems including long QT syndrome, serious arrhythmias, very slow heart rate, or prolonged QTc on ECG
  • Participants who have central nervous system leukemia
  • Participants having serious ongoing medical conditions or infections including uncontrolled infections, severe organ disease, or conditions that make study participation unsafe
  • Participants who are pregnant, breastfeeding, or unwilling to use contraception
  • 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

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Interventions

DRUGQTX-2101 + ATRA

The experimental regimen consists of IV ATO administered once daily during induction, given continuously, for up to a maximum of 60 days. During consolidation, QTX-2101 is administered once daily, per investigator's protocol. ATRA is administered orally in two divided daily doses during induction, given continuously until bone marrow remission (not exceeding 60 days). During consolidation, ATRA is taken orally in two divided daily doses following a 2-weeks-on / 2-weeks-off schedule within each 8-week cycle.

DRUGIV arsenic trioxide (ATO) + ATRA

The comparator regimen consists of IV ATO administered once daily during induction, given continuously, for up to a maximum of 60 days. During consolidation, IV ATO is administered once daily, per investigator's protocol. ATRA is administered orally in two divided daily doses during induction, given continuously until bone marrow remission (not exceeding 60 days). During consolidation, ATRA is taken orally in two divided daily doses following a 2-weeks-on / 2-weeks-off schedule within each 8-week cycle.


Locations(4)

Quetzal Site 1

Duarte, California, United States

Quetzal Site 4

Buffalo, New York, United States

Quetzal Site 2

The Bronx, New York, United States

Quetzal Site 3

Charlottesville, Virginia, United States

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NCT07504458


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