This is a first in human dose escalation trial to determine the safety of administering GPC2 CAR T cells in patients with advanced neuroblastoma or retinoblastoma.
Patients must be ≥ 1 year of age
Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment. Patients who were initially considered low- or intermediate-risk, but then reclassified as high-risk are also eligible.
That is recurrent/relapsed or refractory/persistent according to INRC AND
For which standard curative measures do not exist or are no longer effective.
Patients with active hepatitis B or active hepatitis C.
Patients with HIV infection.
Patients with uncontrolled active infection.
Patients with primary or acquired immunodeficiency disorder.
Sequential
GPC2 CAR T cells
Biological/VaccineThe GPC2 CAR T investigational product is comprised of autologous human T cells that have been genetically modified to express a GPC2-targeting chimeric antigen receptor (CAR) transgene.
Determine the Maximum Tolerated Dose of GPC2 CAR T cells
The Maximum Tolerated Dose of GPC2 CAR T cells will be determined by measuring the incidence of dose limiting toxicities following administration of the product.
Frequency of Adverse Events Following GPC2 CAR T cell administration
Assess the frequency and severity of treatment related adverse events following administration of GPC2 CAR T cells.
Manufacturing Feasibility of GPC2 CAR T cells
Manufacturing Feasibility will be evaluated as the Percentage of patients with GPC2 CAR T cell products that meet release criteria
Persistence of GPC2 CAR T cells
Persistence of GPC2 CAR T cells will be measured by Polymerase Chain Reaction (or flow cytometry) analysis of whole blood to detect and quantify survival of GPC2 CAR T cells over time.
Preliminarily define the clinical activity of GPC2 CAR T in patients with relapsed or refractory neuroblastoma or metastatic retinoblastoma
Overall Response Rate will be determined based on international Neuroblastoma Response Criteria or the COG Retinoblastoma ARET0321/ Response and Response Assessment in Pediatric Neuro-Oncology.
Severity of Adverse Events Following GPC2 CAR T cell administration.
The safety of GPC2 CAR T cell therapy reinfusions will be measured by the monitoring the frequency and severity of adverse events after multipleGPC2 CAR T cells infusions.
Click on the lead sponsor to view all of their trials.
Stephan Grupp MD PhD
Lead sponsor
Children's Hospital of Philadelphia
Sponsor institution
Children's Hospital of Philadelphia
Collaborator
Gilead Sciences
Collaborator
University of Pennsylvania
Collaborator
National Cancer Institute (NCI)
Collaborator
Kite, A Gilead Company
Collaborator
