A Phase 1/2, Open-label Study Evaluating the Efficacy, Safety, and Pharmacokinetics (PK) of Luveltamab Tazevibulin (STRO-002) in Infants and Children < 12 Years of Age With CBFA2T3::GLIS2 Acute Myeloid Leukemia (AML)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 24
- 试验地点
- 36
- 主要终点
- Evaluate efficacy of luveltamab tazevibulin monotherapy
研究概览
简要总结
This trial will evaluate whether luveltamab tazevibulin is well tolerated and active against a rare form of AML carrying a particular genetic abnormality called CBFA2T3::GLIS2 that arises in infants and children. To be treated in this trial children must have a leukemia which did not respond or recurred after prior treatment. Luveltamab tazevibulin is an antibody-drug conjugate, which brings tazevibulin, an anticancer drug, to a molecule called FOLR1, present on the surface of CBFA2T3::GLIS2 AML cells.
详细描述
This is a registrational international, multicenter, two-part open label Phase 1/2 trial in an extremely rare pediatric disease (around 17 new patients a year in US and 10 in EU). Part 1 randomizes subjects 1:1 to one of two luveltamab tazevibulin dose cohorts (1a and 1b). Part 2 further evaluates the safety and the efficacy of the selected dose. Subjects who achieve complete remission after two cycles of treatment may continue luveltamab tazevibulin as monotherapy, while non-responders at PI discretion may add luveltamab tazevibulin with standard of care (SOC) AML treatments. Luveltamab tazevibulin is given IV every two week as monotherapy and every 4 weeks when given with chemotherapy.
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
- 1 Day 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •AML with CBFA2T3::GLIS2 gene fusion centrally confirmed
- •Refractory or relapsed disease with ≥ 5% bone marrow involvement with leukemic blasts by morphology
- •Age < 12 years.
- •Lansky performance of ≥ 50
- •Adequate organ functions
排除标准
- •Active central nervous system (CNS) disease (CNS3)
- •Pre-existing clinically significant corneal disorders or constitutional diseases associated with an increased risk of AML treatment toxicities
- •Active or uncontrolled infections or other active severe intercurrent illnesses,
- •Prior treatment with a FOLR1- targeting ADCs or with ADCs that contain a tubulin inhibitor
- •History of allogeneic hematopoietic stem cell transplant or any organ transplant in the prior 84 days
- •Graft versus host disease (GVHD) of any grade or GVHD treatment with exception of low dose steroids
研究组 & 干预措施
Cohort 1
Luveltamab tazevibulin 3.5mg every 2 weeks
干预措施: Luveltamab tazevibulin (Drug)
Cohort 2
Luveltamab tazevibulin 4.3 mg every 2 weeks
干预措施: Luveltamab tazevibulin (Drug)
结局指标
主要结局
Evaluate efficacy of luveltamab tazevibulin monotherapy
时间窗: Up to 12 weeks
Complete remission rate
次要结局
- Assess additional efficacy outcome measures(Up to 2 years)
- Evaluate safety measures(Up to 2 years)
- To characterize the PK of luveltamab tazevibulin(Up to 2 years)
- Assess the immunogenic potential of luveltamab tazevibulin(Up to 2 years)