GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.

Sponsor
Chinese PLA General Hospital
Study ID
NCT07090538
Phase
EARLY_PHASE1
Status
Not Yet Recruiting

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Conditions

Eligibility Criteria

Sex
ALL
Age
6 Years - 12 Years
Healthy Volunteers
Not accepted

Interventions

  • Eliglustat — DRUG
    Drug: GM-CSF + Eliglustat Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and GSL Synthetase Inhibitor

Study Details

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.

Key Dates

Start date
Aug 1, 2025
Status verified
Jul 2025
Primary completion
Aug 31, 2026
Completion
Aug 31, 2027

Study Design

Enrollment
10 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: GM-CSF + Eliglustat
    Eliglustat 42mg will be administered twice daily in patients in the first 14 days and the following every other week until 24 weeks. For patients who still benefit from the trial, eliglustat 84mg will be administered twice daily every other week to 96 weeks. Prior to the initiation of Eliglustat, GM-CSF was administered for a period of five days at a dose of 250 µg/m2/day. Subsequent to this, GM-CSF is administered at a dosage of 500 µg/m2/day on Days 1-5.

Primary Outcome Measure

Objective response rate (ORR) [ Time Frame: Up to 120 days after the last dose of study drugs ]

Central Contacts

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