Clinical trial · Interventional
GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.
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.
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neuroblastoma | Neuroblastoma | ONTOLOGY_EXACT | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Eliglustat | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- GM-CSF + Eliglustat
- description
- 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.
- interventionNames
- Drug: Eliglustat
Primary outcomes (1)
- measure
- Objective response rate (ORR)
- timeFrame
- Up to 120 days after the last dose of study drugs
- description
- Objective response rate includes complete response and partial response defined by investigators according to RECIST 1.1or iRECIST criteria.
Secondary outcomes (2)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 Years
- Maximum age
- 12 Years
Show eligibility criteria text
Inclusion Criteria: 1. 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. 2. The participant is at least 6years old at time of enrollment. 3. At least one measurable lesion at baseline according to RECIST version 1.1. 4. 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. 5. 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. 6. Eastern Cooperative Oncology Group (ECOG) performance status score≤2 and Estimated life expectancy of more than 3 months. 7. Patient(and/ or their parent/ legal guardian) is willing to participate and able to provide signed informed consent. Exclusion Criteria: 1. Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF 2. Existing major organ dysfunction \> Grade 2, with the exception of hearing loss, hematological status, kidney and liver function 3. Active life-threatening infection 4. History of allergy or intolerance to study drug components. 5. The participant is CYP2D6 ultra-rapid metabolizer. 6. The participant is known to be allergy to Eliglustat. 7. The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity.
References
Publications (0)
Data not yet available