NCT05642455招募中1 期
A Phase 1/2 Open Label, Basket Study to Assess the Safety, Tolerability and Anti-Tumor Activity of Afamitresgene Autoleucel in Pediatric Subjects With MAGE-A4 Positive Tumors
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- USWM CT, LLC
- 入组人数
- 20
- 试验地点
- 11
- 主要终点
- Incidence, duration, and severity of Treatment Emergent Adverse Events as assessed by Investigator Evaluation.
研究概览
简要总结
This is a pediatric basket study to investigate the safety and efficacy of afamitresgene autoleucel in HLA-A*02 eligible and MAGE-A4 positive subjects aged 2-17 years of age with advanced cancers.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has histologically confirmed diagnosis of any one of the following cancers: (A) Synovial Sarcoma (SS), (B) MPNST, (C) Neuroblastoma, or (D) Osteosarcoma (OS).
- •(A) Synovial Sarcoma: 2 to 17 years (B) MPNST, Neuroblastoma and Osteosarcoma: 2 to 21 years
- •Body weight ≥ 10 kg
- •Must have previously received a systemic chemotherapy
- •Measurable disease prior to lymphodepletion according to RECIST v1.1 (or INCR, 2017 Neuroblastoma only).
- •HLA-A*02 positive
- •Tumor shows MAGE-A4 expression confirmed by central laboratory.
- •Performance Status:
- •(A) Subjects ≥16: Eastern Cooperative Oncology Group (ECOG) 0 or 1 (B) Subjects 2 to 16: Lansky score ≥ 80
- •Subject has anticipated life expectancy of greater than 3 months in the opinion of the investigator.
排除标准
- •Positive for HLA-A*02:05 in either allele; or any A*02 having same protein sequence as HLA-A*02:05
- •History of allergic reactions attributed to compounds of similar chemical or biologic composition to fludarabine, cyclophosphamide.
- •History of autoimmune or immune mediated disease
- •Known central nervous system (CNS) metastases.
- •Other prior malignancy that is not considered by the Investigator to be in complete remission
- •Clinically significant cardiovascular disease
- •Active infection with human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or human T cell leukemia virus
- •Pregnant or breastfeeding
- •Experiencing ongoing rapid disease progression that in the opinion of the Investigator significantly increases the subjects risk associated with treatment.
研究组 & 干预措施
Afamitresgene autoleucel
Experimental
干预措施: Afamitresgene autoleucel (Genetic)
结局指标
主要结局
Incidence, duration, and severity of Treatment Emergent Adverse Events as assessed by Investigator Evaluation.
时间窗: 3.5 years
Determination of incidence, severity and duration of adverse events * Incidence of dose limiting toxicities DLTs * AEs including serious adverse events (SAEs) * Incidence, severity, and duration of the AEs of special interest * Replication competent lentivirus (RCL) * T-cell clonality and insertional oncogenesis (IO)
次要结局
- Efficacy: Objective response rate (ORR) assessed by investigator per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (or by International Neuroblastoma Response Criteria [INRC] 2017 in Neuroblastoma subjects)(3.5 years)
- Time to response (TTR)(3.5 years)
- Duration of Response (DoR)(3.5 years)
- Best overall response (BOR)(3.5 years)
- Progression Free Survival (PFS)(3.5 years)
- Overall Survival (OS)(15 years)
- Characterize the in vivo cellular pharmacokinetics (PK) profile of afamitresgene autoleucel by evaluation of PBMC samples for peak persistence.(3.5 years)
- Development and validation of an invitro diagnostic (IVD) assay for the screening of tumor antigen expression for regulatory approval.(3.5 years)
研究者
研究点 (11)
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