NeuroblastomaPhase 2NCT03033303

A Study of the Effect of Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin in Patients in First Remission of High-Risk Neuroblastoma

A Phase 2 study testing Hu3F8, GM-CSF and Isotretinoin in people with Neuroblastoma, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The purpose of this study is to test see the combined effects of the study drug called Humanized 3F8 (Hu3F8) when used with granulocyte-macrophage colony stimulating factor (GM-CSF). Hu3F8 plus GM-CSF could prevent your neuroblastoma from growing, but it could also cause side effects. Read more about this study

Who is it for?: Any age
Conditions the study is for: Neuroblastoma
What is being tested?: Hu3F8, GM-CSF and Isotretinoin
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 59 participants
Where: 1 siteUnited States
Study dates: 23 Jan 2017to 29 Sep 2025
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
Patients must have high-risk NB (MYCN-amplified stage 2/3/4/4S of any age and MYCN-nonamplified stage 4 in patients greater than 18 months of age).
Patients must be in first CR/VGPR
Patients must have a negative human anti-hu3F8 antibody (HAHA) titer

Who cannot join

Existing major organ dysfunction, i.e., renal., cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3, with the exception of hearing loss and hematologic toxicity.
Active life-threatening infection.
Inability to comply with protocol requirements

Trial locations and contacts

1 site in 1 country.

United States1 site

Memorial Sloan Kettering Cancer Center
New York, New York, 10065

Other trials in United States

About this study

Official title: Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin for Consolidation of First Remission of Patients With High-Risk Neuroblastoma: A Phase II Study

Conditions: Neuroblastoma
Being tested: Hu3F8, GM-CSF, Isotretinoin
Keywords: Hu3F8, GM-CSF, Isotretinoin, 16-1643
Medical subject headings: Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Hu3F8Biological
Day 1: hu3F8 infused iv over ~30 to 90 minutes. Day 3: hu3F8 infused iv over ~30 to 90 minutes. Day 5: hu3F8 infused iv over ~30 to 90 minutes.
GM-CSFDrug
Days -4 to 0: GM-CSF 250 mcg/m2/day, subcutaneously. Days 1 to 5: GM-CSF 500 mcg/m2/day, subcutaneously.
IsotretinoinDrug
Isotretinoin is administered at 160 mg/m2/d, divided into two doses, x14 days.

Study groups in NCT03033303
Study group	Role	What they receive
Hu3F8/GM-CSF Plus Isotretinoin	Experimental	In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF. Receives: Biological: Hu3F8; Drug: GM-CSF; Drug: Isotretinoin

What is being measured

The primary outcome is the main question the study is designed to answer.

Relapse-free Survival (RFS)
Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.
Measured: 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

23 Jan 201729 Sep 2025

First posted26 Jan 2017 (estimated)
Study start23 Jan 2017
Primary completion29 Sep 2025
Study completion29 Sep 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for NeuroblastomaStudies of the same condition, open and under way.
More trials run by Memorial Sloan Kettering Cancer CenterThe 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: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF.
Masking: None (open label)
Enrolment: 59 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03033303
Sponsor's protocol ID: 16-1643
Status last verified: Sep 2025
First submitted: 24 Jan 2017
Last updated: 30 Oct 2026
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Kushner BH, Modak S, Mauguen A, Basu EM, Kramer K, Roberts SS, Cheung IY, Cheung NV. A Phase II Trial of Naxitamab plus Stepped-up Dosing of GM-CSF for Patients with High-Risk Neuroblastoma in First Complete Remission. Clin Cancer Res. 2025 May 15;31(10):1877-1884. doi: 10.1158/1078-0432.CCR-24-3427.
PubMed 40067131

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
Active, not recruitingPhase 1
GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma
Neuroblastoma

All Neuroblastoma trials

Study at a glance

From the registry record, last updated by the sponsor on 30 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Hu3F8/GM-CSF Immunotherapy Plus Isotretinoin for Consolidation of First Remission of Patients With High-Risk Neuroblastoma: A Phase II Study
ClinicalTrials.gov ID: NCT03033303
Acronym / sponsor ID: 16-1643
Conditions: Neuroblastoma
Keywords: Hu3F8, GM-CSF, Isotretinoin, 16-1643
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 59 participants (actual)
Lead sponsor: Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
Collaborators: Y-mAbs Therapeutics
Study start: 23 Jan 2017 (actual)
Primary completion: 29 Sep 2025 (actual)
Study completion: 29 Sep 2025 (actual)
First posted: 26 Jan 2017 (estimated)
Results: Posted 30 Oct 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: In this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF.

Arm	Type	Receives
Hu3F8/GM-CSF Plus Isotretinoin	Experimental	Biological: Hu3F8, Drug: GM-CSF, Drug: IsotretinoinIn this phase II single arm trial, patients with HR-NB in first CR or VGPR undergo consolidation by using hu3F8/GM-CSF x5 cycles and isotretinoin x6 cycles. Isotretinoin starts after cycle 2 of hu3F8/GM-CSF.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (4 listed)

Diagnosis of NB as defined by a) histopathology (confirmed by the MSK Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
Patients must have high-risk NB (MYCN-amplified stage 2/3/4/4S of any age and MYCN-nonamplified stage 4 in patients greater than 18 months of age).
Patients must be in first CR/VGPR
Patients must have a negative human anti-hu3F8 antibody (HAHA) titer

Key exclusion criteria (3 listed)

Existing major organ dysfunction, i.e., renal., cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3, with the exception of hearing loss and hematologic toxicity.
Active life-threatening infection.
Inability to comply with protocol requirements

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
Hu3F8Day 1: hu3F8 infused iv over ~30 to 90 minutes. Day 3: hu3F8 infused iv over ~30 to 90 minutes. Day 5: hu3F8 infused iv over ~30 to 90 minutes.	Biological	Hu3F8/GM-CSF Plus Isotretinoin
GM-CSFDays -4 to 0: GM-CSF 250 mcg/m2/day, subcutaneously. Days 1 to 5: GM-CSF 500 mcg/m2/day, subcutaneously.	Drug	Hu3F8/GM-CSF Plus Isotretinoin
IsotretinoinIsotretinoin is administered at 160 mg/m2/d, divided into two doses, x14 days.	Drug	Hu3F8/GM-CSF Plus Isotretinoin

Primary outcome

Relapse-free Survival (RFS)
Time frame: 5 years
Patients are considered a response failure under this protocol if progressive disease is evident before two years. Deaths from toxicity attributable to protocol treatment will be counted as events. Patients who withdraw or are lost to followup before two years will be considered as progressions.

Results and safety

Results posted to the registry on 30 Oct 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Hu3F8/GM-CSF Plus Isotretinoin	59	59

Primary outcome: Relapse-free Survival (RFS)

Time frame: 5 years. Median (Full Range). Unit: months

Measure	Hu3F8/GM-CSF Plus Isotretinoin n = 59
Value	10.2 (2.3 to 51.0)

Adverse events

Time frame: 5 years. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Hu3F8/GM-CSF Plus Isotretinoin	6 of 59 (10.2%)	59 of 59 (100%)	10 of 59

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40067131Kushner BH, Modak S, Mauguen A, Basu EM, Kramer K, Roberts SS, Cheung IY, Cheung NV. A Phase II Trial of Naxitamab plus Stepped-up Dosing of GM-CSF for Patients with High-Risk Neuroblastoma in First Complete Remission. Clin Cancer Res. 2025 May 15;31(10):1877-1884. doi: 10.1158/1078-0432.CCR-24-3427.
Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.

Related trials for Neuroblastoma

RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
Active, not recruitingPhase 1
GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma
Neuroblastoma

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. This study is not recruiting at the moment.

Brian H. Kushner, MD principal investigator

Memorial Sloan Kettering Cancer Center

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 Hu3F8, GM-CSF and Isotretinoin in people with Neuroblastoma, run by Memorial Sloan Kettering Cancer Center at 1 site in United States.

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 3 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: finished, as of the sponsor's last update on 30 Oct 2026. 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 United States. 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?

Yes. Results are posted in the registry (first posted 30 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Relapse-free Survival (RFS) (time frame: 5 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03033303, which also lists 1 linked publication.

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 30 Oct 2026; AzTrials last refreshed its copy on 7 Nov 2026.