A Pivotal Open-label Phase 3 Clinical Study Evaluating the Efficacy and Safety of QTX-2101 in Combination With All-trans Retinoic Acid in Newly Diagnosed, Low-risk Acute Promyelocytic Leukemia
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 150
- 试验地点
- 22
- 主要终点
- Maximum observed plasma (concentration (Cmax) of QTX-2101 for ASIII
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 71 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
QTX-2101
QTX-2101 (oral arsenic trioxide; ATO) All-trans-retinoic-acid (ATRA; oral)
干预措施: QTX-2101 + ATRA (Drug)
IV ATO
IV Arsenic Trioxide (ATO) All-trans-retinoic-acid (ATRA; oral)
干预措施: IV arsenic trioxide (ATO) + ATRA (Drug)
结局指标
主要结局
Maximum observed plasma (concentration (Cmax) of QTX-2101 for ASIII
时间窗: Up to 1 cycle of consolidation therapy (each cycle is 8 weeks)
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
时间窗: Up to 60 days of induction and 3 8-week cycles of consolidation treatment
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.
次要结局
- 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)
- EQ-5D-5L domain unit of measure and measurement tool(Up to 10 months)
- Overall Survival(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)(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)