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

NCT07504458 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: QTX-2101-3012025-524810-28-00
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 4

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

Keywords

APL; Acute Promyelocytic Leukemia; Hematology; Oncology; Leukemia; Hematologic Disease; Drug Therapy; Therapeutics; Blood Cancer; Heme Malignancy; Arsenic; low-risk APL; standard-risk APL

Outcome Measures

Primary Outcomes (2)

• Maximum observed plasma (concentration (Cmax) of QTX-2101 for ASIII

  Cmax is defined as the maximum observed plasma concentration following administration of \[investigational product\], determined from plasma concentration-time data.

  Up to 1 cycle of consolidation therapy (each cycle is 8 weeks)

• Molecular complete remission (molecular CR) rate

  mCR is defined as the absence of detectable PML-RARA fusion transcript in bone marrow assessed by a validated quantitative reverse transcription polymerase chain reaction (RT-qPCR) assay .The mCR rate is defined as the proportion of participants achieving molecular remission at the specified assessment time point following induction and consolidation therapy.

  Up to 60 days of induction and 3 8-week cycles of consolidation treatment

Secondary Outcomes (8)

• To characterize the safety and tolerability of QTX-2101/ATRA and IV ATO/ATRA

  Throughout approximately 10 months of study treatment

• To characterize the event-free survival (EFS) of QTX-2101/ATRA

  Assessed for up to 3 years after the first dose of treatment, or until treatment failure (disease progression), death, or study completion, whichever occurs first

• Area under the plasma concentration-time curve (AUC) of QTX-2101 for ASIII

  Up to 10 months

• To complete a model-based concentration QT relationship evaluation

  Up to 10 months

• EORTC QLQ-C30 domain unit of measure and measurement tool

  Up to 10 months

Study Arms (2)

QTX-2101

EXPERIMENTAL

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

Drug: QTX-2101 + ATRA

IV ATO

ACTIVE COMPARATOR

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

Drug: IV arsenic trioxide (ATO) + ATRA

Interventions

QTX-2101 + ATRA DRUG

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.

QTX-2101

IV arsenic trioxide (ATO) + ATRA DRUG

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.

IV ATO

Eligibility Criteria

• Age: 18 Years - 71 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Quetzal Therapeutics: lead

Study Sites (4)

Quetzal Site 1

Duarte, California, 91010, United States

RECRUITING

Quetzal Site 4

Buffalo, New York, 14203, United States

RECRUITING

Quetzal Site 2

The Bronx, New York, 10467, United States

RECRUITING

Quetzal Site 3

Charlottesville, Virginia, 22908, United States

RECRUITING

Related Links

• Oral arsenic trioxide ORH-2014 pharmacokinetic and safety profile in patients with advanced hematologic disorders

MeSH Terms

Conditions

Leukemia, Promyelocytic, Acute; Neoplasms; Leukemia; Hematologic Diseases; Hematologic Neoplasms

Interventions

Tretinoin; Arsenic Trioxide

Condition Hierarchy (Ancestors)

Leukemia, Myeloid, Acute; Leukemia, Myeloid; Neoplasms by Histologic Type; Hemic and Lymphatic Diseases; Neoplasms by Site

Intervention Hierarchy (Ancestors)

Vitamin A; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Diterpenes; Pigments, Biological; Biological Factors; Arsenicals; Inorganic Chemicals; Oxides; Oxygen Compounds

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 16, 2026
• First Posted: March 31, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): December 1, 2030
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)