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GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.
Sponsor: Chinese PLA General Hospital
Summary
This exploratory clinical study will evaluate the efficacy and feasibility of combining a GSL synthase inhibitor with a granulocyte-macrophage colony stimulating factor (GM-CSF) in patients with advanced or metastatic neuroblastoma. Six to eight eligible patients are expected to be treated in this clinical trial: 1) Assessing the anti-tumour effects of GSL synthase inhibitors in combination with immune checkpoint inhibitors and/or GM-CSF; 2) To assess immunological or clinical predictive biomarkers of efficacy and toxicity; and 3) Detecting changes in the tumour microenvironment (TME) and the dynamics of peripheral blood immune cells after treatment with a GSL synthase inhibitor combined with GM-CSF.
Official title: A Single-arm Exploratory Trial of GSL Synthetase Inhibitor Plus Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and/ or Immune Checkpoint Inhibitor in Advanced/Metastatic Neuroblastoma.
Key Details
Gender
All
Age Range
6 Years - 12 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2025-08-01
Completion Date
2027-08-31
Last Updated
2025-07-29
Healthy Volunteers
No
Conditions
Interventions
Eliglustat
Drug: GM-CSF + Eliglustat Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and GSL Synthetase Inhibitor
Locations (2)
Biotherapeutic Department and Hematology Department of Chinese PLA General Hospital
Beijing, China
Department of Pediatrics, The First Medical Center, Chinese PLA General Hospital
Beijing, China