Phase 1 Trial of GPC2-Directed Chimeric Antigen Receptor Autologous T Cells (GPC2 CAR T) for Relapsed or Refractory Neuroblastoma and Metastatic Retinoblastoma
Stephan Grupp MD PhD
Summary
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.
Description
Despite the use of intensive multimodal chemoradiotherapy, surgery, autologous stem cell transplant and GD2-targeted immunotherapy for the treatment of patients with high-risk neuroblastoma, approximately 60% of children still die from this disease and survivors suffer lifelong treatment related comorbidities. Similarly, children with retinoblastoma with extra-ocular metastasis experience a poor prognosis despite the availability of intensive systemic chemotherapy or external beam radiation therapy. Among children with metastatic retinoblastoma treated with intensive multimodality treatment, 3…
Eligibility
- Age range
- 1+ years
- Sex
- All
- Healthy volunteers
- No
Neuroblastoma Inclusion Criteria: 1. Patients must be ≥ 1 year of age 2. 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. 3. Patients must have a previously histologically confirmed diagnosis of neuroblastoma: 1. That is recurrent/relapsed or refractory/persistent according to INRC AND 2. For which standard curative measures do not exist or are no longer effective. 3. patients at first relapse are elig…
Interventions
- BiologicalGPC2 CAR T cells
The 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.
Location
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania