GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated Hi… (NCT07090538) | Clinical Trial Compass
Not Yet RecruitingEarly Phase 1
GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.
China10 participantsStarted 2025-08-01
Plain-language 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.
Who can participate
Age range6 Years – 12 Years
SexALL
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Inclusion criteria
✓. Neuroblastoma must be documented at the point of diagnosis, which is defined as one of the following: histopathology of a biopsy of a solid tumour; bone marrow aspirate or biopsy suggestive of neuroblastoma with elevated blood or urine catecholamine metabolite levels.
✓. The participant is at least 6years old at time of enrollment.
✓. At least one measurable lesion at baseline according to RECIST version 1.1.
✓. Have adequate organ function as assessed by the laboratory required by protocol, which should be confirmed within 2 weeks prior to the first dose of study drugs.
✓. Previous treatment must be completed for more than 4 weeks prior to the enrollment of this study, and subjects have recovered to \<= grade 1 toxicity.
✓. Eastern Cooperative Oncology Group (ECOG) performance status score≤2 and Estimated life expectancy of more than 3 months.
✓. Patient(and/ or their parent/ legal guardian) is willing to participate and able to provide signed informed consent.
Exclusion criteria
What they're measuring
1
Objective response rate (ORR)
Timeframe: Up to 120 days after the last dose of study drugs