Pediatric Brain Tumor Consortium Phase I Study of CD40 Agonist Sotigalimab in Pediatric and Young Adult Patients with Recurrent CNS Tumors and Newly Diagnosed DIPG.
Clin Cancer ResOct 1, 2026
Holly B Lindsay, Catherine A Billups, Mehmet Kocak et al.
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In 30 seconds
The PBTC-051 study evaluated the safety and preliminary efficacy of the CD40 agonist sotigalimab in 31 pediatric and young adult patients with recurrent CNS tumors and newly diagnosed DIPG. This phase I multicenter trial found that while sotigalimab was well-tolerated, no patients achieved an objective response. The recommended phase II dose for stratum 1 was 0.6 mg/kg every 3 weeks.
Key findings
- 6-month progression-free survival for stratum 1 was 13.3% (SE, 8.1%).
- 6-month progression-free survival for stratum 2 was 31.2% (SE, 14.8%).
- 44.4% overall survival was observed in stratum 2 (SE, 16.6%).
- More than 40% of patients developed antidrug antibodies.
Why it matters
This study provides insights into the safety profile and potential efficacy of sotigalimab in a vulnerable pediatric population with challenging CNS tumors, highlighting the need for continued exploration of immunotherapeutic options.