Recruiting

Phase 3

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

Sponsor:

Quetzal Therapeutics

Code:

NCT07504458

Conditions

Acute Promyelocytic Leukemia (APL)

Acute Promyelocytic Leukaemia

Acute Promyelocytic Leukemia With PML-RARA

Acute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA

Acute Promyelocytic Leukemia

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

QTX-2101 + ATRA

IV arsenic trioxide (ATO) + ATRA

Study Details

Brief 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.

Conditions

Acute Promyelocytic Leukemia (APL)

Acute Promyelocytic Leukaemia

Acute Promyelocytic Leukemia With PML-RARA

Acute Promyelocytic Leukemia With t(15;17)(q24.1;q21.2); PML-RARA

Acute Promyelocytic Leukemia

Study ID

NCT07504458

Start date

Jun 8, 2026

Status verified date

Jun, 2026

Completion date

Dec, 2030

Anticipated

Primary completion date

Mar, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

150 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: QTX-2101

QTX-2101 (oral arsenic trioxide; ATO) All-trans-retinoic-acid (ATRA; oral)

active comparator: IV ATO

IV Arsenic Trioxide (ATO) All-trans-retinoic-acid (ATRA; oral)

Interventions

QTX-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.

IV 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.

Primary outcome measure

Maximum observed plasma (concentration (Cmax) of QTX-2101 for ASIII [ Time Frame: Up to 1 cycle of consolidation therapy (each cycle is 8 weeks) ]
Molecular complete remission (molecular CR) rate [ Time Frame: Up to 60 days of induction and 3 8-week cycles of consolidation treatment ]

Central Contacts and Locations

Locations

Quetzal Site 16

Recruiting

Birmingham, Alabama, United States, 35233

Contacts