NCT03373097

Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors

Phase I/II Study of Anti-GD2 Chimeric Antigen Receptor-Expressing T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Sponsor: Bambino Gesù Hospital and Research Institute · Academic / Other
• Sex: All
• Age: 12 Months – 25 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to test the safety and efficacy of GD2-CART01, a CAR T cell treatment targeting GD2 in paediatric or young adult patients with High Risk and/or relapsed/refractory Neuroblastoma. A small exploratory cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included.

Detailed description

The study will consist of 2 phases, a Phase I or dose escalation phase and a Phase II or expansion phase. Paediatric or young adult patients with relapsed High Risk and/or relapsed/refractory Neuroblastoma will be enrolled in the study. After completion of the phase I portion of the study, a small cohort of patients with GD2-positive tumors other than Neuroblastoma has also been included. Eligible patients will undergo leukapheresis in order to harvest T cells, which will be manufactured to obtain the autologous CAR T product GD2-CART01, a GD2-targeting CAR T product. Briefly, the patients will be treated with a lymphodepleting regimen containing conventional chemotherapic agents and subsequently will receive a single infusion of GD2-CART01. Moreover, the product contains a suicide gene safety switch (namely inducible Caspase 9): in case of relevant toxicities, the patient will receive the dimerizing agent in order to activate the apoptotic pathway in the infused T cells. After infusion of CAR T cells, the patients will enter a 5-year active follow-up period.

Conditions

• Neuroblastoma
• Neuroblastoma Recurrent
• GD2-positive Solid Tumors
• Osteosarcoma
• Ewing Sarcoma
• Sarcoma

Interventions

Type	Name	Description
BIOLOGICAL	GD2-CART01	Following a lymphodepleting treatment with conventional chemotherapy, patients will be treated with 1.0 to 10.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells as a single dose.

Timeline

• Start date: 2018-01-05
• Primary completion: 2026-12-01
• Completion: 2027-12-01
• First posted: 2017-12-14
• Last updated: 2025-02-05

Locations

1 site across 1 country: Italy