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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
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.
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, China
RECRUITINGSun Yat-sen University Cancer Center
Guangzhou, China
NOT_YET_RECRUITINGTo evaluate the safety and tolerability of umbilical cord blood natural killer cell injection in combination with other treatments of high-risk, recurrent/refractory neuroblastoma in children
According to the '3+3' design principle, the Maximum Tolerated Dose (MTD) is determined. Adverse events observed during the trial are graded according to the NCI CTCAE 5.0 criteria. Adverse events related to the drug (definitely related, probably related, or possibly related) occurring within 36 days after the first infusion of umbilical cord blood NK cells (during the first cycle) are considered as Dose-Limiting Toxicity (DLT). The definitions of DLT include: (1) Hematological toxicity: Grade 4 neutropenia lasting ≥7 days; Grade 4 thrombocytopenia lasting ≥7 days; (2) Grade 3/4 non-hematological toxicities (excluding nausea, vomiting, and alopecia); (3) Any toxicity leading to a delay in chemotherapy for more than 2 weeks.
Time frame: One weeks after the first cycle (each cycle is 28 days)
overall response rate (ORR) after 2 courses of umbilical cord blood natural killer cell infusion
ORR was evaluated according to the International Neuroblastoma Response Criteria (INRC)
Time frame: Two weeks after the second cycle (a total of 16 weeks after the strart of first umbilical cord blood natural killer cell infusion)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.