NCT06631391

Phase I Study of Umbilical Cord Blood Natural Killer (NK) Cell Therapy for Children With High-risk, R/R Neuroblastoma.

Pilot Study of Umbilical Cord Blood Natural Killer (NK) Cell Therapy for Pediatric Neuroblastoma.

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Sun Yat-sen University · Academic / Other
• Sex: All
• Age: 1 Day – 18 Years
• Healthy volunteers: Not accepted

Summary

Neuroblastoma is the most common extracranial solid tumor, with more than half of the patients diagnosed at the metastatic stage, classified as high-risk. High-risk neuroblastoma has a poor prognosis and low survival rate. Despite treatment with induction, consolidation, and maintenance therapy including GD2 monoclonal antibody, the survival rate is only about 60%, and many patients still relapse, progress, and die. NK cell therapy is an emerging immunotherapy that can effectively inhibit and kill tumor cells without significant adverse reactions, reducing the risk of tumor recurrence and metastasis, and improving patients' immunity and quality of life. Its safety has been widely recognized. Currently, clinical trials of NK cell infusion therapy for neuroblastoma patients are ongoing, and NK cell-based immunotherapy holds great clinical promise for neuroblastoma. We plan to conduct a phase I clinical trial on umbilical cord blood NK cell therapy in combination with other treatments (GD2 antibody, chemotherpay, etc) for high-risk, recurrent/refractory neuroblastoma in children to determine the maximum tolerated dose of umbilical cord blood NK cell therapy in these patients, thereby laying the foundation for future combination therapies and phase II and III clinical studies.

Detailed description

To improve the prognosis of high-risk, Recurrent/Refractory neuroblastoma, we conducted a prospective Phase I clinical trial. Patients with high-risk and Recurrent/Refractory neuroblastoma who had undergone multidisciplinary treatment received umbilical cord blood NK cell infusions. The safety and efficacy of umbilical cord blood NK cells in combination with other therapy (GD2 antibody, chemotherpay, etc) were evaluated.

Conditions

• Neuroblastoma

Interventions

Type	Name	Description
BIOLOGICAL	umbilical cord blood NK cells	This is a phase I prospective clinical trial, including phase Ia and phase Ib. Phase Ia include 3 dose levels, utilizing a 3+3 design principle, and each dose level will enroll at least 3 subjects. The recommended dose for phase Ia was used in phase Ib. Each patient receives two courses of umbilical cord blood NK cell therapy (a total of 8 infusions of umbilical cord blood NK cells) and other therapy.

Timeline

• Start date: 2024-11-01
• Primary completion: 2026-11-30
• Completion: 2029-11-30
• First posted: 2024-10-08
• Last updated: 2025-02-20

Locations

2 sites across 1 country: China