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.
Subject has histologically confirmed diagnosis of any one of the following cancers: (A) Synovial Sarcoma (SS), (B) MPNST, (C) Neuroblastoma, or (D) Osteosarcoma (OS).
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).
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.
Single group
Afamitresgene autoleucel
GeneticSingle infusion of afamitresgene autoleucel Dose: For subjects ≥10 kg to \<40 kg: starting dose of 0.025 - 0.200 x 10'9 transduced cells/kg. For subjects ≥40 kg 1.0x109 to 10x109 transduced by a single intravenous infusion
Incidence, duration, and severity of Treatment Emergent Adverse Events as assessed by Investigator Evaluation.
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)
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)
ORR is defined as incidence of complete responses or partial responses as assessed by RECIST v1.1 or INRC, 2017
Time to response (TTR)
For patients who are observed to respond to afamitresgene autoleucel in the time from date of infusion to achieve a partial response or complete response (TTR) is assessed
Duration of Response (DoR)
For patients who are observed to respond to afamitresgene autoleucel the DoR is the date of initial response (including confirmation) from date of infusion up until disease progression
Best overall response (BOR)
BOR is assessed by the investigator per RECIST V1.1 or INCR, 2017 (for Neuroblastoma subjects)
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