Neuroblastoma RecurrentPhase 2NCT05754684
Quadruple Immunotherapy for Neuroblastoma
A Phase 2 study testing Natural killer cell, Dinutuximab beta and Interleukin-2 in people with Neuroblastoma Recurrent, run by Hong Kong Children's Hospital at 1 site in Hong Kong.
Trial summary
This is a single-arm clinical trial to evaluate the efficacy and safety of quadruple immunotherapy with natural killer (NK) cells, anti-GD2 antibody, cytokines (interleukin-2 (IL-2) and granulocyte-macrophage colony stimulating factor (GM-CSF)) and retinoid X receptor gamma (RXRg) agonist spironolactone for paediatric… Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Neuroblastoma Recurrent
- What is being tested?
- Natural killer cell, Dinutuximab beta and Interleukin-2
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteHong Kong
- Study dates
- 1 Jan 2022to 31 Dec 2025
- Phase
- Phase 2
- 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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- relapsed or refractory neuroblastoma
- Adequate organ function: creatinine clearance ≥40 ml/min/1.73m2, total bilirubin ≤3 times upper limit of normal and ALT ≤500 IU/L, left ventricular shortening fraction ≥25%, and oxygen saturation ≥92% in room air
- Karnofsky or Lansky performance status score ≥50
- Has an appropriate HLA-haploidentical NK-cell donor available
Who cannot join
- Pregnant or lactating woman
- HIV infection
- Patients for whom conventional treatment is deemed more appropriate
- Patients who are unlikely to benefit, e.g., terminal malignancy with life expectancy <1 month
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Hong Kong1 site, 1 recruiting
- Recruiting
Hong Kong Children's Hospital
Hong Kong
Daniel Cheuk, 852-35136049, cheukkld@gmail.com
About this study
Official title: Quadruple Immunotherapy for Paediatric Patients With Relapsed or Refractory Neuroblastoma
This is a single-arm clinical trial to evaluate the efficacy and safety of quadruple immunotherapy with natural killer (NK) cells, anti-GD2 antibody, cytokines (interleukin-2 (IL-2) and granulocyte-macrophage colony stimulating factor (GM-CSF)) and retinoid X receptor gamma (RXRg) agonist spironolactone for paediatric patients with relapsed or refractory neuroblastoma.
Read the detailed description
Included patients will receive intravenous infusion of donor NK cells on day 0, and anti-GD2 antibody (dinutuximab) on day -6 to day -2. IL-2 will be given subcutaneously on day -1, day +1, day +3, day +5, day +7, and day +9. Subcutaneous injection of GM-CSF will be started on day 0, given daily till neutrophil count >1000/mm3. Spironolactone will be started orally on day -1, given three times daily till cessation of GM-CSF.
Alternative anti-GD2 antibody (Naxitamab) can be used instead of dinutuximab, to be given on day -5, day -3, day +1 and day +3.
- Conditions
- Neuroblastoma Recurrent
- Being tested
- Natural killer cell, Dinutuximab beta, Interleukin-2, Granulocyte-Macrophage Colony-Stimulating Factor, Spironolactone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Natural killer cellBiological
Also called NK cell
Natural killer cells isolated from HLA-haploidentical relative donor
Dinutuximab betaDrug
Also called Ch14.18
Dinutuximab beta iv for 5 days
Interleukin-2Drug
Also called Aldesleukin
Interleukin-2 sc alternate day for 6 doses
Granulocyte-Macrophage Colony-Stimulating FactorDrug
Also called Sargramostim
Granulocyte-macrophage colony-stimulating factor sc daily till ANC >2,000/mm3
SpironolactoneDrug
Also called Aldactone
Spironolactone po three time daily
NaxitamabDrug
Also called hu3F8
Naxitamab iv for 4 days (as alternative for dinutuximab)
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | Quadruple immunotherapy Receives: Biological: Natural killer cell; Drug: Dinutuximab beta; Drug: Interleukin-2; Drug: Granulocyte-Macrophage Colony-Stimulating Factor; Drug: Spironolactone; Drug: Naxitamab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients who have objective response in the tumor
Objective response = complete response + partial response + minor response + stable disease
Measured: 1-2 months
Secondary outcomes (5)
Overall survival at 1 year
From the date of treatment start until the date of death from any cause, assessed up to 1 year
Measured: up to 1 year
Progression-free survival
From date of treatment start until the date of first documented progression or the date of death from any cause, whichever comes first, assessed up to 1 year
Measured: up to 1 year
Proportion of patients who have tumor relapse
relapse = reappearance of tumor after complete response
Measured: up to 1 year
Number of patients who experience adverse events
Adverse events are graded according to Cancer Therapy Evaluation Program Common Terminology Criteria for Adverse Events version 5 (CTCAEv5)
Measured: up to 1 month
Percentage of donor NK cells
Percentage of donor natural killer cells in the recipient's blood will be evaluated weekly from the date of NK cell infusion for 4 weeks, then every 2 weeks till donor NK cells are undetectable
Measured: up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2023
- Study start1 Jan 2022
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuroblastoma RecurrentStudies of the same condition, open and under way.
- 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hong Kong Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- The University of Hong Kong
- Responsible party
- Principal investigator, Cheuk Ka Leung Daniel, Hong Kong Children's Hospital
- Investigators
- Daniel Cheuk, Principal investigator, Hong Kong Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05754684
- Sponsor's protocol ID
- HKCH-REC-2021-007
- Status last verified
- Mar 2025
- First submitted
- 18 Nov 2022
- Last updated
- 15 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Quadruple Immunotherapy for Paediatric Patients With Relapsed or Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT05754684
- Acronym / sponsor ID
- HKCH-REC-2021-007
- Conditions
- Neuroblastoma Recurrent
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- Hong Kong Children's HospitalUniversity, hospital or other
- Collaborators
- The University of Hong Kong
- Study start
- 1 Jan 2022 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 6 Mar 2023 (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 is a single-arm clinical trial to evaluate the efficacy and safety of quadruple immunotherapy with natural killer (NK) cells, anti-GD2 antibody, cytokines (interleukin-2 (IL-2) and granulocyte-macrophage colony stimulating factor (GM-CSF)) and retinoid X receptor gamma (RXRg) agonist spironolactone for paediatric patients with relapsed or refractory neuroblastoma.
Detailed description
Included patients will receive intravenous infusion of donor NK cells on day 0, and anti-GD2 antibody (dinutuximab) on day -6 to day -2. IL-2 will be given subcutaneously on day -1, day +1, day +3, day +5, day +7, and day +9. Subcutaneous injection of GM-CSF will be started on day 0, given daily till neutrophil count >1000/mm3. Spironolactone will be started orally on day -1, given three times daily till cessation of GM-CSF.
Alternative anti-GD2 antibody (Naxitamab) can be used instead of dinutuximab, to be given on day -5, day -3, day +1 and day +3.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention | Experimental | Biological: Natural killer cell, Drug: Dinutuximab beta, Drug: Interleukin-2, Drug: Granulocyte-Macrophage Colony-Stimulating Factor, Drug: Spironolactone, Drug: NaxitamabQuadruple immunotherapy |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- relapsed or refractory neuroblastoma
- Adequate organ function: creatinine clearance ≥40 ml/min/1.73m2, total bilirubin ≤3 times upper limit of normal and ALT ≤500 IU/L, left ventricular shortening fraction ≥25%, and oxygen saturation ≥92% in room air
- Karnofsky or Lansky performance status score ≥50
- Has an appropriate HLA-haploidentical NK-cell donor available
Key exclusion criteria (4 listed)
- Pregnant or lactating woman
- HIV infection
- Patients for whom conventional treatment is deemed more appropriate
- Patients who are unlikely to benefit, e.g., terminal malignancy with life expectancy <1 month
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 |
|---|---|---|
| Natural killer cellAlso: NK cellNatural killer cells isolated from HLA-haploidentical relative donor | Biological | Intervention |
| Dinutuximab betaAlso: Ch14.18Dinutuximab beta iv for 5 days | Drug | Intervention |
| Interleukin-2Also: AldesleukinInterleukin-2 sc alternate day for 6 doses | Drug | Intervention |
| Granulocyte-Macrophage Colony-Stimulating FactorAlso: SargramostimGranulocyte-macrophage colony-stimulating factor sc daily till ANC >2,000/mm3 | Drug | Intervention |
| SpironolactoneAlso: AldactoneSpironolactone po three time daily | Drug | Intervention |
| NaxitamabAlso: hu3F8Naxitamab iv for 4 days (as alternative for dinutuximab) | Drug | Intervention |
Primary outcome
Proportion of patients who have objective response in the tumor
Time frame: 1-2 months
Objective response = complete response + partial response + minor response + stable disease
5 secondary outcomes
Overall survival at 1 year
Time frame: up to 1 year
Progression-free survival
Time frame: up to 1 year
Proportion of patients who have tumor relapse
Time frame: up to 1 year
Number of patients who experience adverse events
Time frame: up to 1 month
Percentage of donor NK cells
Time frame: up to 1 year
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 15 Sep 2025.
- Individual participant dataSharing planned: No
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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.
Daniel Cheuk principal investigator
Hong Kong Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Natural killer cell, Dinutuximab beta and Interleukin-2 in people with Neuroblastoma Recurrent, run by Hong Kong Children's Hospital at 1 site in Hong Kong.
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 4 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 15 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Hong Kong. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Daniel Cheuk (852-35136049, cheukkld@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of patients who have objective response in the tumor (time frame: 1-2 months). 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 15 Sep 2025.