NCT03373071

Anti-CD19 CAR T Cells in Pediatric Patients Affected by Relapsed/Refractory CD19+ ALL and NHL

Phase I/II Study of Anti-CD19 Chimeric Antigen Receptor-Expressing T Cells in Pediatric Patients Affected by Relapsed/Refractory CD19+ Acute Lymphoblastic Leukemia and Non Hodgkin Lymphoma

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Bambino Gesù Hospital and Research Institute · Academic / Other
• Sex: All
• Age: 6 Months – 25 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this phase I study is to evaluate the safety and to establish the recommended dose of CD19-CART01 infused in pediatric patients affected by relapsed/refractory B-ALL or NHL with measurable Bone Marrow (BM) involvement. The phase II extension is aimed at testing the efficacy of the treatment at the optimal dose defined in the phase I.

Detailed description

The study will consist of 2 phases, a Phase I or dose escalation phase and a Phase II or expansion phase. Paediatric/young adult patients with relapsed or refractory B cell ALL will be enrolled. Eligible patients will undergo leukapheresis in order to harvest T cells, which is the starting material for the manufacture. Autologous CAR T product directed against CD19-expressing tumor cells (CD19-CART01) will be produced and, after a lymphodepletion with conventional chemoterapic agents, the patient will receive CD19-CART01 intravenously. The construct contains also the suicide gene safety switch "inducible Caspase 9"; therefore, in case of relevant toxicities, the patient will receive the dimerizing agent in order to induce the apoptosis of the cells. After the treatment, the patients will then enter a 36-month follow-up period.

Conditions

• CD19-ALL
• CD19-LNH

Interventions

Type	Name	Description
BIOLOGICAL	CD19-CAR T cell	Following lymphodepletion with chemotherapy (cyclophosphamide and fludarabine) patients will be treated with 0.5 to 3.0 x 10⁶/kg CD19 Chimeric Antigen Receptor (CAR) positive T cells as a single dose. In case of toxicity, the patient will receive the dimerizing drug activating the suicide safety switch in order to improve the safety of the treatment

Timeline

• Start date: 2017-12-23
• Primary completion: 2021-05-11
• Completion: 2025-01-24
• First posted: 2017-12-14
• Last updated: 2025-02-05

Locations

1 site across 1 country: Italy