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
Summary
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.
Arms & interventions
- GeneticAfamitresgene autoleucel
Single 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
Outcome measures
Primary
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)
Time frame: 3.5 years
Secondary
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)
Time frame: 3.5 years
Time to response (TTR)
Time frame: 3.5 years
Duration of Response (DoR)
Time frame: 3.5 years
Best overall response (BOR)
Time frame: 3.5 years
Progression Free Survival (PFS)
Time frame: 3.5 years
Overall Survival (OS)
Time frame: 15 years
Characterize the in vivo cellular pharmacokinetics (PK) profile of afamitresgene autoleucel by evaluation of PBMC samples for peak persistence.
Time frame: 3.5 years
Development and validation of an invitro diagnostic (IVD) assay for the screening of tumor antigen expression for regulatory approval.
Time frame: 3.5 years
Eligibility criteria
Study locations (10)
Stanford University
Palo Alto, California, 94305
National Institutes of Health
Bethesda, Maryland, 20892
Dana Farber Cancer Institute
Boston, Massachusetts, 10065
Washington University
St Louis, Missouri, 63110
Memorial Sloan Kettering Kids
New York, New York, 10065
Duke University School of Medicine
Durham, North Carolina, 27710
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Hospital of Philedephia
Philadelphia, Pennsylvania, 19104
Seattle Children's Hospital
Seattle, Washington, 98105
University of Wisconsin Cancer Center
Madison, Wisconsin, 53715