Phase 1 Trial of PHOX2B Peptide-Centric Chimeric Antigen Receptor Autologous T Cells (PHOX2B PC-CAR T) for Relapsed Neuroblastoma
Stephan Grupp MD PhD
Summary
This is a first in human dose escalation trial to determine the safety of administering PHOX2B PC-CAR T cells in patients with advanced, high-risk neuroblastoma.
Description
Neuroblastoma is a tumor of childhood arising from neural crest-derived cells of the developing sympathetic nervous system. While neuroblastomas in young infants often spontaneously regress, children diagnosed with advanced disease after 18 months of age experience poor overall survival despite intensive therapy. Neuroblastoma is a heterogenous disease, but approximately 50% of patients have a "high-risk" clinical phenotype defined by well-established clinical signs and molecular biomarkers. Over 50% of patients ultimately relapse and survivors are often burdened with significant long-term th…
Eligibility
- Age range
- 1+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Patients must be ≥ 1 years of age 2. Patients must demonstrate expression of at least one of the following HLA alleles by HLA genotyping (conducted at CHOP) to be eligible: HLA-A\*24:02, HLA-A\*24:03, HLA-A\*24:04, HLA-A\*24:07, HLA-A\*24:124, HLA-A\*24:143, HLA-A\*24:17, HLA-A\*24:242, HLA-A\*24:305, HLA-A\*24:314, HLA-A\*24:33, HLA-A\*24:353, HLA-A\*24:41, HLA-A\*24:51, HLA-A\*24:63, HLA-A\*24:87, HLA-A\*24:92, HLA-A\*23:01, HLA-A\*23:17, HLA-A\*23:25, HLA-A\*23:39, 3. Disease Status A. Patients must have high-risk neuroblastoma according to COG risk classificatio…
Interventions
- BiologicalPHOX2B PC-CAR T Cells
The PHOX2B PC-CAR T cell investigational product is comprised of autologous human T cells that have been genetically modified to express a a PHOX2B-targeting chimeric antigen receptor (CAR) transgene.
Location
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania