GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.
Condition(s) studied
Investigational drug(s) / intervention(s)
Eliglustat: Drug: GM-CSF + Eliglustat Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and GSL Synthetase Inhibitor
Study 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.
Eligibility
Primary outcome measure(s)
- Objective response rate (ORR) — Up to 120 days after the last dose of study drugs
Objective response rate includes complete response and partial response defined by investigators according to RECIST 1.1or iRECIST criteria.
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
More Chinese PLA General Hospital trials in China
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07090538 on ClinicalTrials.gov ↗ ← All trials in China