NCT07090538Not yet recruitingEarly phase 1
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 · Last updated 29 Jul 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 21 Jul 2025
- Start date
- 1 Aug 2025
- Primary completion date
- 31 Aug 2026
- Lead sponsor
- Chinese PLA General Hospital
- Diseases
- Neuroblastoma, Childhood cancers
- NCT number
- NCT07090538
What you commit to
- Study type: Interventional · Early phase 1
- Planned participants: 10
- From 1 Aug 2025 to the primary completion date, 31 Aug 2026
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF
- The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity.
Other illness
- Active life-threatening infection
- History of allergy or intolerance to study drug components.
- The participant is known to be allergy to Eliglustat.
Other
- Existing major organ dysfunction \> Grade 2, with the exception of hearing loss, hematological status, kidney and liver function
- The participant is CYP2D6 ultra-rapid metabolizer.
Who the study is looking for
- 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.
Age in the registry: 6 years to 12 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Department of Pediatrics, The First Medical Center, Chinese PLA General Hospital Coordinator of this site? Confirm its status | Beijing | China | Unknown status |
| Biotherapeutic Department and Hematology Department of Chinese PLA General Hospital Coordinator of this site? Confirm its status | Beijing | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.