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

NCT07090538 | Not Yet Recruiting Early Phase 1

• 1 other identifier: CHN-PLAGH-ELI-721
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief 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.

Conditions

Neuroblastoma

Keywords

GSL synthetase inhibitor; GM-SCF

Outcome Measures

Primary Outcomes (1)

• Objective response rate (ORR)

  Objective response rate includes complete response and partial response defined by investigators according to RECIST 1.1or iRECIST criteria.

  Up to 120 days after the last dose of study drugs

Secondary Outcomes (2)

• Progression Free Survival (PFS)

  Up to 2 years

• Overall Survival (OS)

  Up to 2 years

Other Outcomes (2)

• Immunological response (cytokines, lymphocyte phenotype)

  Up to 120 days after the last dose of study drugs

• Biomarkers predictive of response and toxicity

  Up to 120 days after the last dose of study drugs

Study Arms (1)

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.

Drug: Eliglustat

Interventions

Eliglustat DRUG

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

Also known as: Cerdelga

GM-CSF + Eliglustat

Eligibility Criteria

• Age: 6 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chinese PLA General Hospital: lead

Study Sites (2)

Biotherapeutic Department and Hematology Department of Chinese PLA General Hospital

Beijing, 100853, China

Location

Department of Pediatrics, The First Medical Center, Chinese PLA General Hospital

Beijing, China

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

eliglustat

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Weidong Han

  Biotherapeutic Department, Chinese PLA General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Weidong Han, Ph.D

CONTACT

010-66937231; hanwdrsw@sina.com

Chen Feng

CONTACT

01055499217; 301yisheng@sohu.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Biotherapeutic Department

Study Record Dates

• First Submitted: July 21, 2025
• First Posted: July 29, 2025
• Study Start: August 1, 2025
• Primary Completion (Estimated): August 31, 2026
• Study Completion (Estimated): August 31, 2027
• Last Updated: July 29, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

CN (2)