Unituxin (dinutuximab)

P1, N=27, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Mar 2026 --> Mar 2027

P3, N=1449, Completed, National Cancer Institute (NCI) | Active, not recruiting --> Completed

P1/2, N=76, Not yet recruiting, National Cancer Institute (NCI)

P2, N=94, Active, not recruiting, Children's Oncology Group | Trial completion date: Apr 2026 --> Mar 2029

The GADEKILL γδ T and NK cells were analyzed by flow cytometry for the expression of activating and inhibitory receptors and for cytotoxicity against NB, both with and without dinutuximab-β, at a 1:1 effector-to-target ratio...Finally, NB cell lysis positively correlated with B7H6 and BTN2A1, and B7H6-blocking experiments revealed a significant decrease in target cell lysis when cells highly expressing B7H6 were used as targets. Our study demonstrated the potential antineuroblastoma activity of the GADEKILL, supporting its therapeutic use, particularly in the context of relapsed/refractory R/R HR-NB with low GD2 expression.

Recent advances in immunotherapy have reshaped the treatment landscape, with anti-GD2 monoclonal antibodies such as dinutuximab and naxitamab demonstrating significant clinical benefit, especially when combined with granulocyte-macrophage colony-stimulating factor (GM-CSF)...This review synthesizes current evidence on immunotherapeutic strategies in neuroblastoma, highlighting resistance pathways, biomarker-driven approaches, and the evolving clinical trial landscape. Future directions emphasize personalized, biomarker-guided immunotherapy to improve efficacy, reduce toxicity, and establish durable, curative outcomes for children with neuroblastoma.

P1, N=11, Active, not recruiting, Children's Hospital Los Angeles | Recruiting --> Active, not recruiting

For patients ≥547 days old, any age cut-off ≤40 months discriminated younger (superior EFS/OS) versus older patients; no cut-off was optimal. OCAYA diagnosed 2010-2022 (post-immunotherapy era) had superior outcomes versus 2003-2009. Stratification by comprehensive molecular biomarkers will likely best inform novel therapeutic strategies for OCAYA.

P1, N=24, Recruiting, Emory University | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026

P2, N=118, Not yet recruiting, New Approaches to Neuroblastoma Therapy Consortium

Dinutuximab, approved for high-risk neuroblastoma, improved event-free survival when combined with cytokines with standard therapy alone (NCT00026312). Similarly, Naxitamab, in combination with GM-CSF, achieved an overall response rate in relapsed/refractory neuroblastoma (NCT03363373)...Additionally, Racotumomab (anti-NeuGcGM3) demonstrated a survival benefit in advanced non-small cell lung cancer, extending median overall survival compared to placebo (NCT01240447). Despite these advances, challenges remain in improving patient selection, reducing off-target toxicities such as neuropathic pain, and addressing resistance mechanisms. This review examines the latest developments in ganglioside-mediated cancer therapy, emphasizing current clinical outcomes, highlighting the need for more precise targeting approaches, and exploring rational combination strategies to enhance therapeutic efficacy.

P2, N=62, Recruiting, New Approaches to Neuroblastoma Therapy Consortium | Active, not recruiting --> Recruiting