Arsenic trioxide for Acute promyelocytic leukemia

Inclusion criteria

• {"criterion_text":"- Participants must be ≥18 to <71 years old at the time of signing an informed consent."}
• {"criterion_text":"- Participants must be capable of giving signed and dated institutional review board or independent ethics committee approved informed consent."}
• {"criterion_text":"- Participants must have a diagnosis of APL characterized by the presence of the t(15;17) translocation by fluorescence in situ hybridization or cytogenetics, or PML/RARA gene expression via RT-qPCR."}
• {"criterion_text":"- Participants must be classified as low- or intermediate-risk APL, defined as WBC count ≤10×109/L at diagnosis."}
• {"criterion_text":"- PART 1 Only: Participants with LR-APL should have completed induction and 3 cycles of Consolidation treatment with IV ATO and ATRA. They should have documented mCR at the time of study entry."}
• {"criterion_text":"- PART 2 Only: Participants should be ND LR-APL."}
• {"criterion_text":"- Participants must have organ function as defined below: • Serum total bilirubin ≤3.0 mg/dL • Alanine aminotransferase and Aspartate aminotransferase ≤3× upper limit of normal • Creatinine clearance ≥30 mL/min"}
• {"criterion_text":"- Participants must have an Eastern Cooperative Oncology Group Performance Status of ≤2."}
• {"criterion_text":"- Participants must have a serum or high-sensitivity urine pregnancy test (for females of childbearing potential) that is negative at the Screening Visit and immediately prior to initiation of treatment (first dose of study intervention)."}
• {"criterion_text":"- Participants must be willing and able to comply with the scheduled study visits, treatment plans, including receipt of study intervention at the study site through the end of study period, laboratory tests, contraception guidance, and other procedures. Note: Participants must be willing and able to provide written informed consent and commit to complete the full treatment and follow-up procedures as outlined in the protocol, unless discontinuation is medically indicated or required by the investigator or sponsor."}

Exclusion criteria

• {"criterion_text":"- Participants who have had treatment for APL with ATRA for >7 days prior to the first dose of study intervention (Part 2 only)."}
• {"criterion_text":"- Participants with a history of other cancers must not be receiving active treatment (with radiation or chemotherapy) and must be free of disease for 2 years prior to the Screening Visit with the exception of localized prostate cancer treated with hormone monotherapy, breast cancer treated with hormone monotherapy, basal cell carcinoma, nonmelanoma skin cancer, or cervical carcinoma in situ."}
• {"criterion_text":"- Participants with an active, life-threatening, or clinically important uncontrolled systemic infection requiring hospitalization."}
• {"criterion_text":"- Participants who have a known malabsorption syndrome or other condition that may impair absorption of study medication (eg, gastrectomy) or who are unable to swallow oral medication."}
• {"criterion_text":"- Participants who have other severe acute or chronic medical conditions (and/or psychiatric conditions or laboratory abnormalities) that may increase the expected risk to the participant (ie, the risk associated with the study participation or study intervention administration) or that may interfere with the interpretation of study results or, in the judgment of the investigator, would make the participant inappropriate for entry into this study."}
• {"criterion_text":"- Immunocompromised participants with increased risk of opportunistic infections, including known human immunodeficiency virus (HIV)-positive participants with CD4 counts ≤350 cells/mm3 or history of opportunistic infection in the last 12 months. To ensure that effective antiretroviral therapy, when used in eligible HIV-positive participants, is tolerated and that toxicities are not confusing with investigational drug toxicities, participants should be on an established antiretroviral therapy for at least 4 weeks and have an HIV viral load less than 400 copies/mL prior to the Screening Visit."}
• {"criterion_text":"- Participants who have a known active or chronic hepatitis B or active hepatitis C virus (HCV) infection. Participants with a history of HCV infection who have completed curative therapy for HCV at least 12 weeks before the Screening Visit and have a documented undetectable viral load at the Screening Visit are eligible for randomization."}
• {"criterion_text":"- Participants with indications requiring uninterrupted anticoagulation (eg, mechanical heart valves) due to increased risk of hemorrhagic complications during induction (Part 2)."}
• {"criterion_text":"- Pregnant females, breastfeeding females, and males not willing to comply with contraceptive requirements or females of childbearing potential not willing to comply with contraceptive requirements."}
• {"criterion_text":"- Participants who are likely to withdraw consent at the completion of treatment, or during study follow-up, for the sole purpose of enrolling in another interventional clinical study for APL or another investigational therapy, unless recommended by the investigator or sponsor."}
• {"criterion_text":"- Participants who have suspected central nervous system involvement with leukemia."}
• {"criterion_text":"- Participants with Grade ≥2 neuropathy."}
• {"criterion_text":"- Participants with a history of torsade de pointes."}
• {"criterion_text":"- Participants with ECG abnormalities, including: • Congenital long QT syndrome. • History or presence of significant ventricular or atrial tachyarrhythmia. • Clinically significant resting bradycardia (<50 beats per minute). • Corrected QT interval (QTc) >450 msec on screening ECG using QTcF, obtained as the mean from 3 QTcF values from a triplicate standard resting ECG at screening. • Right bundle branch block plus left anterior hemiblock, bifascicular block."}
• {"criterion_text":"- Participants with unresolved related AEs from prior exposure of IV ATO (Part 1 only) unless the AEs are Grade 1 or completely resolved prior to enrollment."}
• {"criterion_text":"- Participants with a current or recent (within 3 months prior to the Screening Visit) history of symptomatic congestive heart failure."}
• {"criterion_text":"- Participants who have a known contraindication or hypersensitivity to ATO, ATRA, or any of their excipients, including participants with hypersensitivity to soy and/or peanut (ATRA)."}
• {"criterion_text":"- Participants who received any other investigational agents within 30 days of the Screening Visit or <5 half-lives since completion of previous investigational therapy, whichever is shorter."}

Primary endpoints

• {"endpoint_text":"- PK parameters, including maximum plasma concentration (Cmax) and area under curve (AUC) for all metabolites of arsenic trioxide, including arsenious acid (AsIII), arsenic acid (ASV), total arsenic, dimethylarsinic acid (DMA), and monomethylarsenic acid (MMA).","definition_or_measurement_approach":"PK parameters measured including Cmax and AUC for specified arsenic trioxide metabolites; measured via plasma sampling and PK analysis (population PK analyses referenced in secondary endpoints)."}
• {"endpoint_text":"- Molecular complete remission (mCR) defined as absence of promyelocytic leukemia/ retinoic acid receptor alpha (PML/RARA) in the bone marrow per European Leukemia Net (ELN) 2019 criteria for APL. Negativity of PML/RARA is defined as PML/RARA transcript level below 10-4, confirmed by centralized quantitative reverse transcription-polymerase chain reaction (RTqPCR) testing using an assay with a limit of detection (LOD) of 10-5.","definition_or_measurement_approach":"mCR defined per ELN 2019: absence of PML/RARA in bone marrow; negativity = transcript level <10^-4 confirmed by centralized RT-qPCR assay with LOD 10^-5."}

Secondary endpoints

• {"endpoint_text":"- Incidence of adverse events (AEs), clinically significant laboratory changes, significant electrocardiogram (ECG) findings, and vital sign changes.","definition_or_measurement_approach":"Safety assessed by AE reporting, laboratory tests, ECG findings and vital signs per protocol-specified schedules."}
• {"endpoint_text":"- Landmark EFS at the time of the primary endpoint analysis, defined as time from randomization to the date of induction treatment failure (failure to achieve complete remission [CR]/morphologic complete remission [CRi] after induction), no achievement of mCR after 3 consolidation courses, relapse after CR/mCR, or death from any cause, whichever comes first.","definition_or_measurement_approach":"Event-free survival measured from randomization to induction failure, failure to achieve mCR after 3 consolidations, relapse after CR/mCR, or death; landmark analysis at primary endpoint analysis timepoint."}
• {"endpoint_text":"- Other parameters of efficacy, including CR/CRi rate, defined as achieving CR/CRi at the end of induction and end of Consolidation Cycle 3 per ELN 2019 criteria for APL and landmark overall survival (OS), defined as duration from randomization to date of death due to any cause","definition_or_measurement_approach":"CR/CRi assessed per ELN 2019 at end of induction and after Consolidation Cycle 3; OS measured from randomization to death."}
• {"endpoint_text":"- Incidences of AEs, clinically significant laboratory changes, significant ECG, and vital sign changes","definition_or_measurement_approach":"As per safety monitoring; AE incidence and clinically significant changes recorded during study."}
• {"endpoint_text":"- Summary statistics of QTX-2101 steady-state AUC and Cmax, determined by population PK analysis.","definition_or_measurement_approach":"Population PK analysis to determine steady-state AUC and Cmax summary statistics."}
• {"endpoint_text":"- Triplicate ECGs with time-matched PK sampling analysis.","definition_or_measurement_approach":"ECGs performed in triplicate with time-matched PK samples to evaluate concentration–QT relationship."}
• {"endpoint_text":"- Participant-reported outcomes assessing quality of life (eg, EORTC QLQ-C30), treatment convenience and satisfaction (eg, CCSQ), health utility (eg, EQ-5D-5L), and overall treatment burden (eg, number of infusion visits, catheter use, and time in treatment)","definition_or_measurement_approach":"PRO instruments (EORTC QLQ-C30, CCSQ, EQ-5D-5L) and treatment-burden metrics (infusion visits, catheter use, time in treatment) collected per schedule."}

France

Earliest CTIS Part Ii Submission Date

27-04-2026

Latest Decision Or Authorization Date

13-05-2026

Processing Time Days

16

Number Of Sites

5

Number Of Participants

12

Primary sponsor

Full Name

Quetzal Therapeutics LLC

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

CliniChain B.V.

Responsibilities

Ancillary supplies/ Temperature monitoring device

Name

Frontage Laboratories Inc.

Responsibilities

Samples storage and analysis

Name

Almac Clinical Services Limited

Name

Suvoda LLC

Third parties

• {"country":"United States","full_name":"Sensitech Inc.","duties_or_roles":"Temperature monitoring solutions","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Concierge - travel support","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Labconnect LLC","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Quest Diagnostics Inc.","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Clario","duties_or_roles":"eCOA, ECG`","organisation_type":"Health care"}
• {"country":"United States","full_name":"Frontage Laboratories Inc.","duties_or_roles":"Samples storage and analysis","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"CliniChain B.V.","duties_or_roles":"Ancillary supplies/ Temperature monitoring device","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}