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

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.

Timeline

Start

2018-01-05

Primary completion

2026-12

Completion

2027-12

Publications

• Locatelli F, Pagliara D, De Ioris MA, Becilli M, Del Baldo G, Serra A, Mastronuzzi A, Cefalo MG, Li Pira G, Leone G, Bertaina V, Fabozzi F, Di Nardo M, Rosignoli C, D'Andrea ML, Crocoli A, Vennarini S, Sinibaldi M, Di Cecca S, Guercio M, Iaffaldano L, Lucarelli B, Algeri M, Merli P, Colafati GS, De Angelis B, Quintarelli C, Del Bufalo F. GD2-targeting CAR T cells in high-risk neuroblastoma: a phase 1/2 trial. Nat Med. 2025 Nov;31(11):3689-3699. doi: 10.1038/s41591-025-03874-6. Epub 2025 Aug 21.
• Yeku OO, Longo DL. CAR T Cells for Neuroblastoma. N Engl J Med. 2023 Apr 6;388(14):1328-1331. doi: 10.1056/NEJMe2300317. No abstract available.
• Del Bufalo F, De Angelis B, Caruana I, Del Baldo G, De Ioris MA, Serra A, Mastronuzzi A, Cefalo MG, Pagliara D, Amicucci M, Li Pira G, Leone G, Bertaina V, Sinibaldi M, Di Cecca S, Guercio M, Abbaszadeh Z, Iaffaldano L, Gunetti M, Iacovelli S, Bugianesi R, Macchia S, Algeri M, Merli P, Galaverna F, Abbas R, Garganese MC, Villani MF, Colafati GS, Bonetti F, Rabusin M, Perruccio K, Folsi V, Quintarelli C, Locatelli F; Precision Medicine Team-IRCCS Ospedale Pediatrico Bambino Gesu. GD2-CART01 for Relapsed or Refractory High-Risk Neuroblastoma. N Engl J Med. 2023 Apr 6;388(14):1284-1295. doi: 10.1056/NEJMoa2210859.
• Tumino N, Weber G, Besi F, Del Bufalo F, Bertaina V, Paci P, Quatrini L, Antonucci L, Sinibaldi M, Quintarelli C, Maggi E, De Angelis B, Locatelli F, Moretta L, Vacca P, Caruana I. Polymorphonuclear myeloid-derived suppressor cells impair the anti-tumor efficacy of GD2.CAR T-cells in patients with neuroblastoma. J Hematol Oncol. 2021 Nov 12;14(1):191. doi: 10.1186/s13045-021-01193-0.

Drugs

Indications