Open and recent trials

A single-center, open-label Phase-I study of autologous CAR-T cells targeting L1CAM in pediatric patients with relapsed L1CAM-expressing solid tumors. The study uses a 3+3 dose-escalation design with planned expansion cohorts at the recommended Phase-II dose. Cellular kinetics, persistence, and correlative immune profiling are core secondary endpoints. Eligibility includes confirmed L1CAM expression on archived or fresh tumor tissue.

A multi-center, open-label, single-arm Phase-I study assessing the safety, tolerability, and recommended Phase-II dose of larotrectinib combined with selinexor in children with relapsed or refractory neuroblastoma. The study uses a continual reassessment design adapted for pediatric accrual constraints. Correlative endpoints include circulating tumor DNA dynamics and matched organoid drug-response profiling for consenting participants.

A two-part Phase-I/II study evaluating the combination of an oral MEK inhibitor with a CDK4/6 inhibitor in pediatric AT/RT. Part A is a dose-finding cohort in patients with relapsed disease; Part B is a Phase-II expansion in newly diagnosed patients, integrated with maintenance therapy after standard induction. Matched patient-derived organoids are derived at enrollment when tissue is available and used to characterise mechanisms of response.

A multi-center Phase-I/II study evaluating the safety and preliminary activity of anti-GD2 antibody therapy combined with PD-1 checkpoint inhibition in children with high-risk relapsed neuroblastoma. Enrollment has closed; participants remain in active follow-up and survival ascertainment continues for the protocol-defined window. A correlative analysis of paired pre- and on-treatment biopsies is ongoing.

A completed Phase-I dose-finding study of a small-molecule EED inhibitor in pediatric and young-adult patients with relapsed or refractory Ewing sarcoma. The study established a recommended Phase-II dose and characterised pharmacodynamic effects in paired biopsies. Final outcomes were published in 2025; a successor Phase-I/II combination study is in protocol development.