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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
150
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.
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.
Quetzal Site 1
Duarte, California, United States
RECRUITINGQuetzal Site 4
Buffalo, New York, United States
RECRUITINGQuetzal Site 2
The Bronx, New York, United States
RECRUITINGQuetzal Site 3
Charlottesville, Virginia, United States
RECRUITINGMaximum 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.
Time frame: 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.
Time frame: Up to 60 days of induction and 3 8-week cycles of consolidation treatment
To characterize the safety and tolerability of QTX-2101/ATRA and IV ATO/ATRA
Treatment emergent adverse events
Time frame: Throughout approximately 10 months of study treatment
To characterize the event-free survival (EFS) of QTX-2101/ATRA
Time frame: 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
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.
Time frame: Up to 10 months
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
Time frame: Up to 10 months
EORTC QLQ-C30 domain unit of measure and measurement tool
Change from baseline in EORTC QLQ-C30 global health status (0-100 scale)
Time frame: Up to 10 months
EQ-5D-5L domain unit of measure and measurement tool
Change from baseline in EQ-5D-5L index score (0-100)
Time frame: Up to 10 months
Overall Survival
OS is defined as the time from randomization to death from any cause. Participants who are alive at the time of analysis will be censored at the date last known to be alive.
Time frame: 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
Event Free Survival (EFS)
EFS is defined as the time from randomization to the first occurrence of any of the following events: failure to achieve hematologic or molecular remission, relapse (hematologic or molecular), or death from any cause. Participants without an event at the time of analysis will be censored at the date of last adequate disease assessment.
Time frame: 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
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