Symptoms and General Pathology

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Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neuroectodermal Tumors, Primitive, Peripheral

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Neuroepithelial

Neoplasms, Germ Cell and Embryonal

Neoplasms, Glandular and Epithelial

Neoplasms, Nerve Tissue

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Soft Tissue Sarcoma

Ewing Sarcoma

Gaucher Disease Type 2

Neuroepithelioma

After a lymphodepleting regimen the patients will receive 1.0 to 10.0 x 10⁶/kg GD2 Chimeric Antigen Receptor (CAR) positive T cells.

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.

GD2-CART01

Franco Locatelli, MD, PhD

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.

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.

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

Toxicity will be assessed according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) scale, version 4 and the number of patients experiencing DLT will be evaluated

Assessment of Best Overall Response (BOR)

Detection of infused CAR T cell in the peripheral and bone marrow blood

Assessment of the seric cytokines profile

Tumor response assessment through the two different criteria

Assessment of the kinetic of CAR T cell elimination after infusion of the dimerizer

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors	NCT03373097	Neuroblastoma Neuroblastoma R... GD2-positive So... Osteosarcoma Ewing Sarcoma Sarcoma	GD2-CART01	12 Months - 25 Years	Bambino Gesù Hospital and Research Institute
Anti-GD2 CAR T Cells in Pediatric Patients Affected by High Risk and/or Relapsed/Refractory Neuroblastoma or Other GD2-positive Solid Tumors	NCT03373097	Neuroblastoma Neuroblastoma R... GD2-positive So... Osteosarcoma Ewing Sarcoma Sarcoma	GD2-CART01	12 Months - 25 Years	Bambino Gesù Hospital and Research Institute

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