NeuroblastomaEarly Phase 1NCT07090538
GSL Synthetase Inhibitor Plus GM-CSF and/ or Immune Checkpoint Inhibitor in Previously Treated High-Risk Neuroblastoma.
A Early Phase 1 study testing Eliglustat in people with Neuroblastoma, run by Chinese PLA General Hospital at 2 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 12 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Eliglustat
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesChina
- Study dates
- 1 Aug 2025to 31 Aug 2027
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF
- Existing major organ dysfunction > Grade 2, with the exception of hearing loss, hematological status, kidney and liver function
- Active life-threatening infection
- History of allergy or intolerance to study drug components.
- The participant is CYP2D6 ultra-rapid metabolizer.
- The participant is known to be allergy to Eliglustat.
- The participant use drugs that will strongly inhibit CYP2D6 or CYP3A activity.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites
Biotherapeutic Department and Hematology Department of Chinese PLA General Hospital
Beijing, 100853
Department of Pediatrics, The First Medical Center, Chinese PLA General Hospital
Beijing
Feng Chen, Ph.D, 0086-01055499217, 301yisheng@sohu.com
About this study
Official title: 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.
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.
Read the detailed description
Neuroblastoma is the most common extracranial solid tumour in infants and children. It is also known as an immunocold tumour due to the poor infiltration of immune cells in the tumour microenvironment. Tumour-associated macrophages and myeloid-derived suppressor cells account for a significant proportion of neuroblastoma cases and predominantly exert a pro-tumourigenic effect due to the tumour-suppressive microenvironment. Anti-phagocytic signals expressed by tumour cells, such as CD47, inhibit the direct cytophagy of macrophages. Patients in the high-risk group are less immunogenic and therefore less responsive to immune-checkpoint inhibitor (ICI) monotherapy than those in the low- and intermediate-risk groups. A combination of approaches may therefore be required for the immunotherapy of neuroblastoma.
Aberrant expression of glycosphingolipid (GSL) synthase is a fundamental feature of most tumours and tumour microenvironments. Neurological tumours, including neuroblastoma, have been observed to exhibit elevated levels of acidic glycosphingolipids on their surfaces. A number of studies have indicated that there is an increase in the expression of GSL synthase in tumours that are resistant to chemotherapy. Eliglustat, an oral GlcCer synthase inhibitor, has been approved for the treatment of Gaucher disease type 1. Researchers have demonstrated the excellent safety and efficacy of Eliglustat in combination with immune checkpoint inhibitors for treating advanced/metastatic solid tumours and relapsed/refractory haematological malignancies.
For these reasons, an exploratory clinical study was designed to observe the efficacy and feasibility of GSL synthase inhibitors in combination with GM-CSF in the treatment of patients with advanced/metastatic neuroblastoma. The objective of this study was to provide a high level of evidence-based rationale for the combination treatment regimen in these patients. The investigators will administer a combination of Eliglustat and GM-CSF to six patients diagnosed with advanced or metastatic NB. The primary objective of this study is to assess the efficacy and feasibility of Eliglustat in combination with GM-CSF in the treatment of neuroblastoma. The secondary objectives are to assess immunological or clinically predictive biomarkers of efficacy and toxicity, alterations in the tumour microenvironment, and changes in the dynamics of immune cells in the peripheral blood.
- Conditions
- Neuroblastoma
- Being tested
- Eliglustat
- Keywords
- GSL synthetase inhibitor, GM-SCF
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EliglustatDrug
Also called Cerdelga
Drug: GM-CSF + Eliglustat Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and GSL Synthetase Inhibitor
| Study group | Role | What they receive |
|---|---|---|
| GM-CSF + Eliglustat | Experimental | 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. Receives: Drug: Eliglustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate (ORR)
Objective response rate includes complete response and partial response defined by investigators according to RECIST 1.1or iRECIST criteria.
Measured: Up to 120 days after the last dose of study drugs
Secondary outcomes (2)
Progression Free Survival (PFS)
Time from the date of first administration of the study drug to disease progression or death from any cause (any earliest date).
Measured: Up to 2 years
Overall Survival (OS)
Time from the date of first administration of the study drug to the date of death.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2025
- Study start1 Aug 2025 (estimated)
- Primary completion31 Aug 2026 (estimated)
- Study completion31 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Chinese PLA General HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chinese PLA General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Han weidong, Chinese PLA General Hospital
- Investigators
- Weidong Han, Principal investigator, Biotherapeutic Department, Chinese PLA General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07090538
- Sponsor's protocol ID
- CHN-PLAGH-ELI-721
- Status last verified
- Jul 2025
- First submitted
- 21 Jul 2025
- Last updated
- 29 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT07090538
- Acronym / sponsor ID
- CHN-PLAGH-ELI-721
- Conditions
- Neuroblastoma
- Keywords
- GSL synthetase inhibitor, GM-SCF
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Chinese PLA General HospitalUniversity, hospital or other
- Study start
- 1 Aug 2025 (estimated)
- Primary completion
- 31 Aug 2026 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 29 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Neuroblastoma is the most common extracranial solid tumour in infants and children. It is also known as an immunocold tumour due to the poor infiltration of immune cells in the tumour microenvironment. Tumour-associated macrophages and myeloid-derived suppressor cells account for a significant proportion of neuroblastoma cases and predominantly exert a pro-tumourigenic effect due to the tumour-suppressive microenvironment. Anti-phagocytic signals expressed by tumour cells, such as CD47, inhibit the direct cytophagy of macrophages. Patients in the high-risk group are less immunogenic and therefore less responsive to immune-checkpoint inhibitor (ICI) monotherapy than those in the low- and intermediate-risk groups. A combination of approaches may therefore be required for the immunotherapy of neuroblastoma.
Aberrant expression of glycosphingolipid (GSL) synthase is a fundamental feature of most tumours and tumour microenvironments. Neurological tumours, including neuroblastoma, have been observed to exhibit elevated levels of acidic glycosphingolipids on their surfaces. A number of studies have indicated that there is an increase in the expression of GSL synthase in tumours that are resistant to chemotherapy. Eliglustat, an oral GlcCer synthase inhibitor, has been approved for the treatment of Gaucher disease type 1. Researchers have demonstrated the excellent safety and efficacy of Eliglustat in combination with immune checkpoint inhibitors for treating advanced/metastatic solid tumours and relapsed/refractory haematological malignancies.
For these reasons, an exploratory clinical study was designed to observe the efficacy and feasibility of GSL synthase inhibitors in combination with GM-CSF in the treatment of patients with advanced/metastatic neuroblastoma. The objective of this study was to provide a high level of evidence-based rationale for the combination treatment regimen in these patients. The investigators will administer a combination of Eliglustat and GM-CSF to six patients diagnosed with advanced or metastatic NB. The primary objective of this study is to assess the efficacy and feasibility of Eliglustat in combination with GM-CSF in the treatment of neuroblastoma. The secondary objectives are to assess immunological or clinically predictive biomarkers of efficacy and toxicity, alterations in the tumour microenvironment, and changes in the dynamics of immune cells in the peripheral blood.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GM-CSF + Eliglustat | Experimental | Drug: EliglustatEliglustat 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. |
Who may qualify
- Ages
- 6 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (7 listed)
- Any systemic anti-cancer therapy, including chemotherapy or immunotherapy, within 3 weeks before 1st dose of GM-CSF
- Existing major organ dysfunction > Grade 2, with the exception of hearing loss, hematological status, kidney and liver function
- Active life-threatening infection
- History of allergy or intolerance to study drug components.
- The participant is CYP2D6 ultra-rapid metabolizer.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EliglustatAlso: CerdelgaDrug: GM-CSF + Eliglustat Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and GSL Synthetase Inhibitor | Drug | GM-CSF + Eliglustat |
Primary outcome
Objective response rate (ORR)
Time frame: 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.
2 secondary outcomes
Progression Free Survival (PFS)
Time frame: Up to 2 years
Overall Survival (OS)
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2025.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Weidong Han principal investigator
Biotherapeutic Department, Chinese PLA General Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Eliglustat in people with Neuroblastoma, run by Chinese PLA General Hospital at 2 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 29 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Weidong Han, Ph.D (010-66937231, hanwdrsw@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate (ORR) (time frame: Up to 120 days after the last dose of study drugs). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2025.