Neuroblastoma (NB)Early Phase 1NCT03189706
Study of Chemoimmunotherapy for High-Risk Neuroblastoma
A Early Phase 1 study testing Irinotecan, temozolomide and Hu3F8 in people with Neuroblastoma (NB), run by Memorial Sloan Kettering Cancer Center at 1 site in United States.
Trial summary
The purpose of this study is to find out whether an experimental drug called Hu3F8 can be given with the chemotherapy drugs irinotecan and temozolomide and another drug called GM-CSF. The investigators want to find out if this combination is safe and what effect it has on the participant and the disease. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neuroblastoma (NB)
- What is being tested?
- Irinotecan, temozolomide and Hu3F8
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Jun 2017to Jun 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of NB as defined by international criteria,.e., histopathology (confirmed by the MSK Department of Pathology) or bone marrow metastases plus high urine catecholamine levels
- High-risk NB as defined as any of the following:
- Stage 4 with MYCN amplification (any age)
- Stage 4 without MYCN amplification (>1.5 years of age)
- Stage 3 with MYCN amplification (unresectable; any age)
- Stage 4S with MYCN amplification (any age)
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- Patients fulfill one of the following criteria:
- Have evidence of soft tissue disease OR
- If they only have osteomedullary disease at protocol enrollment, they should have:
- Had previously received Hu3F8+GMCSF therapy AND have had less than a complete response to it OR
- Had progressed progressive disease after their most recent anti-neuroblastoma therapeutic regimen
- Patients must have evaluable (microscopic marrow metastasis, elevated tumor markers, positive MIBG or PET scans) or measurable (CT, MRI) disease documented after completion of prior systemic therapy.
- Prior treatment with murine and hu3F8 is allowed.
- Prior treatment with irinotecan or temozolomide is permitted.
- Patients with prior m3F8, hu3F8, ch14.18 or hu14.18 treatment must have a negative HAHA antibody titer. Human anti-mouse antibody positivity is allowed.
- Signed informed consent indicating awareness of the investigational nature of this program.
Who cannot join
- Patients with CR/VGPR disease
- Existing severe major organ dysfunction, i.e., renal, cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3 except for hearing loss, alopecia, anorexia, nausea, and hypomagnesemia from TPN, which may be grade 3
- ANC < 500/uL
- Platelet count <30K/uL
- History of allergy to mouse proteins
- Active life-threatening infection
- Inability to comply with protocol requirements
- Women who are pregnant or breast-feeding
Trial locations and contacts
1 site in 1 country.
United States1 site
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: Phase II Study of Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) Chemoimmunotherapy for High-Risk Neuroblastoma
The purpose of this study is to find out whether an experimental drug called Hu3F8 can be given with the chemotherapy drugs irinotecan and temozolomide and another drug called GM-CSF. The investigators want to find out if this combination is safe and what effect it has on the participant and the disease.
- Conditions
- Neuroblastoma (NB)
- Being tested
- Irinotecan, temozolomide, Hu3F8, GM-CSF
- Keywords
- Hu3F8, Irinotecan, Temozolomide, Sargramostim, Chemoimmunotherapy, 17-251
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IrinotecanDrug
50mg/m^2/day IV will be administered from day 1-5
temozolomideDrug
(given concurrently with Irinotecan) 150mg/m^2/day orally
Hu3F8Biological
2.25mg/kg IV will be administered on days 2, 4, 8 and 10
GM-CSFDrug
250mcg/m2/day SC will be administered on days 6-10
| Study group | Role | What they receive |
|---|---|---|
| Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) | Experimental | Each cycle consists of four doses of hu3F8, five doses each of irinotecan and temozolomide and five doses of GM-CSF. Receives: Drug: Irinotecan; Drug: temozolomide; Biological: Hu3F8; Drug: GM-CSF |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
The regimen will be considered safe if there are no toxicities requiring discontinuation of therapy in at least 9/10 patients during the first two cycles.
Measured: 2 years
response rate (CR+PR)
Response assessment will be based on the best response over the course of four cycles. Disease response for NB will use the International NB Response Criteria. Patients who withdraw from the study prior to cycle 4 with < partial response will also not be considered evaluable for response and will be replaced.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2017
- Study start12 Jun 2017
- Primary completionJun 2027 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuroblastoma (NB)Studies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is a pilot study of HITS in patients with resistant NB.
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Y-mAbs Therapeutics
- Responsible party
- Sponsor
- Investigators
- Shakeel Modak, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03189706
- Sponsor's protocol ID
- 17-251
- Status last verified
- Jul 2026
- First submitted
- 13 Jun 2017
- Last updated
- 6 Jul 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) Chemoimmunotherapy for High-Risk Neuroblastoma
- ClinicalTrials.gov ID
- NCT03189706
- Acronym / sponsor ID
- 17-251
- Conditions
- Neuroblastoma (NB)
- Keywords
- Hu3F8, Irinotecan, Temozolomide, Sargramostim, Chemoimmunotherapy, 17-251
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Y-mAbs Therapeutics
- Study start
- 12 Jun 2017 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 16 Jun 2017 (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.
The purpose of this study is to find out whether an experimental drug called Hu3F8 can be given with the chemotherapy drugs irinotecan and temozolomide and another drug called GM-CSF. The investigators want to find out if this combination is safe and what effect it has on the participant and the disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is a pilot study of HITS in patients with resistant NB.
| Arm | Type | Receives |
|---|---|---|
| Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) | Experimental | Drug: Irinotecan, Drug: temozolomide, Biological: Hu3F8, Drug: GM-CSFEach cycle consists of four doses of hu3F8, five doses each of irinotecan and temozolomide and five doses of GM-CSF. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (16 listed)
- Diagnosis of NB as defined by international criteria,.e., histopathology (confirmed by the MSK Department of Pathology) or bone marrow metastases plus high urine catecholamine levels
- High-risk NB as defined as any of the following:
- Patients fulfill one of the following criteria:
- Patients must have evaluable (microscopic marrow metastasis, elevated tumor markers, positive MIBG or PET scans) or measurable (CT, MRI) disease documented after completion of prior systemic therapy.
- Prior treatment with murine and hu3F8 is allowed.
Key exclusion criteria (8 listed)
- Patients with CR/VGPR disease
- Existing severe major organ dysfunction, i.e., renal, cardiac, hepatic, neurologic, pulmonary, or gastrointestinal toxicity ≥ grade 3 except for hearing loss, alopecia, anorexia, nausea, and hypomagnesemia from TPN, which may be grade 3
- ANC < 500/uL
- Platelet count <30K/uL
- History of allergy to mouse proteins
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 |
|---|---|---|
| Irinotecan50mg/m^2/day IV will be administered from day 1-5 | Drug | Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) |
| temozolomide(given concurrently with Irinotecan) 150mg/m^2/day orally | Drug | Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) |
| Hu3F82.25mg/kg IV will be administered on days 2, 4, 8 and 10 | Biological | Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) |
| GM-CSF250mcg/m2/day SC will be administered on days 6-10 | Drug | Hu3F8, Irinotecan/Temozolomide and Sargramostim (HITS) |
Primary outcome
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 2 years
The regimen will be considered safe if there are no toxicities requiring discontinuation of therapy in at least 9/10 patients during the first two cycles.
response rate (CR+PR)
Time frame: 2 years
Response assessment will be based on the best response over the course of four cycles. Disease response for NB will use the International NB Response Criteria. Patients who withdraw from the study prior to cycle 4 with < partial response will also not be considered evaluable for response and will be replaced.
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 6 Jul 2026.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.
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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. This study is not recruiting at the moment.
Shakeel Modak, 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 Early Phase 1 study testing Irinotecan, temozolomide and Hu3F8 in people with Neuroblastoma (NB), 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 16 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 6 Jul 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?
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?
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?
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 (time frame: 2 years). The study lists 2 primary outcomes in all. 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 6 Jul 2026.