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.
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 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
Parallel
QTX-2101 + ATRA
DrugThe 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
DrugThe 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.
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.
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.
To characterize the safety and tolerability of QTX-2101/ATRA and IV ATO/ATRA
Treatment emergent adverse events
To characterize the event-free survival (EFS) of QTX-2101/ATRA
Area under the plasma concentration-time curve (AUC) of QTX-2101 for ASIII
AUC is defined as the area under the plasma concentration-time curve from time zero to the last measurable concentration (AUC₀-t) and/or extrapolated to infinity (AUC₀-∞), calculated using noncompartmental methods.
To complete a model-based concentration QT relationship evaluation
Time-matched difference in change from baseline in QTcF interval between active treatment and placebo, derived from triplicate 12-lead ECGs at each post-dose time point
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