NeuroblastomaPhase 1NCT07027748
Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma
A Phase 1 study testing Naxitamab, Irinotecan and Temozolomide in people with Neuroblastoma and Neuroblastoma Recurrent, run by Steven DuBois, MD at 2 sites in United States.
Trial summary
This research is being done to investigate a treatment regimen of Irinotecan, Temozolomide, and Sargramostin, and an immunotherapy called Naxitamab and whether giving Naxitamab more slowly reduces the side effects for participants with relapsed or refractory neuroblastoma. Read more about this study
- Who is it for?
- Ages 1 year to 30 years
- Conditions the study is for
- Neuroblastoma, Neuroblastoma Recurrent, Relapsed Neuroblastoma
- What is being tested?
- Naxitamab, Irinotecan and Temozolomide
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- 27 Jun 2025to 1 Dec 2027
- Phase
- 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 1 year to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologic Diagnosis: Patients must have had histologic verification of neuroblastoma or ganglioneuroblastoma or demonstration of neuroblastoma cells in the bone marrow with elevated urinary catecholamines [i.e. > 2 x upper limit of normal (ULN)], at the time of initial diagnosis.
- Relapsed or Refractory Disease Patients must have ONE of the following:
- 1) Any prior episode of recurrent high-risk disease following completion of frontline high-risk therapy. Patients may have received other lines of therapy for treatment of recurrent disease prior to enrolling to this trial.
- 2) Prior progressive high-risk disease during frontline high-risk therapy. Patients may have received other lines of therapy for treatment of progressive disease prior to enrolling to this trial.
- 3) Primary resistant/refractory disease (less than partial response by INRC) detected after the conclusion of at least 4 cycles of aggressive multidrug induction chemotherapy on or according to a high-risk neuroblastoma protocol (examples include ANBL0532, ANBL09P1, ANBL12P1, ANBL1531, ANBL2131) that was treated with additional therapy with the goal of improving remission status prior to enrolling to this trial.
- Documentation of Disease: Patients must have at least ONE of the following at the time of enrollment:
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- 1) Measurable tumor on MRI or CT scan. Measurable is defined as ≥ 10 mm in at least one dimension (or 15 mm in short axis for lymph node) on spiral/helical CT or MRI that is MIBG avid or demonstrates increased FDG uptake on PET scan.
- 2) MIBG-avid lesion detected on MIBG scan with positive uptake at a minimum of one site. This site must represent disease recurrence or known refractory disease at a site not previously radiated.
- 3) In patients with known MIBG non-avid disease, FDG-avid lesion detected on FDG- PET scan with positive uptake at a minimum of one site. This site must represent disease recurrence or known refractory disease at a site not previously radiated.
- Of note, patients with isolated bone marrow only disease are NOT eligible for this trial.
- Prior Therapy: Prior lines of anticancer therapy allowed as described in eligibility section above by disease status. Washout periods from prior therapy are as follows:
- Myelosuppressive chemotherapy: Last dose given 14 days prior to enrollment.
- Small molecule targeted therapies (anti-neoplastic agents including retinoids): Last dose given 7 days prior to enrollment.
- Monoclonal antibodies: Last given at least 7 days or 3 half-lives, whichever is longer, prior to enrollment.
- Radiation:
- Craniospinal irradiation: Last fraction received minimum of six weeks prior to enrollment
- All other radiation: Last fraction received minimum of 14 days prior to enrollment
- Hematopoietic stem cell transplant: Date of autologous stem cell infusion following myeloablative chemotherapy must have been a minimum of 12 weeks prior to enrollment. Patients are not eligible post allogeneic stem cell transplant.
- Cellular therapies (including CAR-T cells, NK cells, other related cellular therapies): 21 days from the last cellular therapy infusion prior to enrollment and recovery from all associated toxicities
- 131I-MIBG therapy: Last therapy received a minimum of 6 weeks prior to enrollment.
- Age: Patients 1 - 30 years of age at the time of enrollment are eligible for this study.
- Performance level: Patients must demonstrate adequate performance level as measured by Karnofsky ≥ 70% for patients aged 16 years or older, OR Lansky ≥ 70% for patients younger than 16 years. Please see Appendix A for performance score measurement.
- Participants must meet the following organ and marrow function as defined below:
- Adequate bone marrow function as defined as BOTH of the following:
- Peripheral absolute neutrophil count (ANC) ≥ 750/uL. Must be more than 14 days from last administration of long-acting myeloid stimulating factor (e.g. pegfilgrastim) or 7 days from last administration of short- acting myeloid stimulating factor (e.g. filgrastim or sargramostim)
- Peripheral platelet count ≥ 75,000/uL. Must be without support, defined as at least 7 days from last platelet transfusion and/or platelet stimulating agent.
- Adequate renal function as defined as EITHER of the following:
- Radioisotope GFR ≥ 70ml/min/1.73 m2
- Serum creatinine based on age/sex as follows:
- Age Maximum Serum Creatinine (mg/dL)
- 1 to < 2 years Male 0.6 Female 0.6
- 2 to < 6 years Male 0.8 Female 0.8
- 6 to < 12 years Male 1.0 Female 1.0
- 12 to < 13 years Male 1.2 Female 1.2
- 13 to < 16 years Male 1.5 Female 1.4
- ---≥ 16 years Male 1.7 Female 1.4
- The threshold creatinine values in this Table were derived from the Schwartz formula for estimating GFR.
- Adequate liver function defined as ALL of the following:
- Total bilirubin ≤ 1.5 x ULN for age*
- ALT ≤ 3.0 x ULN for age (≤ 135 U/L). For the purpose of this study, the ULN for ALT is 45 U/L
- Albumin > 3 g/dL --*If patient has known Gilbert syndrome, direct bilirubin should be used to measure liver function instead of total bilirubin. Direct bilirubin must be within normal limits for age for these patients.
- Adequate cardiac function measured by echocardiogram as defined as EITHER of the following:
- Shortening fraction of ≥ 27%
- Ejection fraction of ≥ 50%
- Adequate blood pressure as defined by BOTH of the following:
- Patients must have < Grade 2 hypertension AND
- Be on no more than one standing antihypertensive
- Adequate pulmonary function: Patients must have adequate pulmonary function, defined as:
- No dyspnea at rest
- No exercise intolerance
- Room air O2 saturation >94%
- Not on chronic oxygen therapy
- Adequate pancreatic function, defined as lipase < 1.5 x ULN
- Able to comply with protocol requirements
- Adequate contraception: The effects of naxitamab on the developing human fetus are unknown. For this reason and because other therapeutic agents used in this trial are known to be teratogenic, participants with potential to become pregnant or to impregnate a partner must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a participant become pregnant or suspect they are pregnant while they or their partner is participating in this study, they should inform the treating physician immediately. Patients treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of protocol therapy.
- Ability to understand and/or the willingness of their parent or legally authorized representative to sign a written informed consent document.
Who cannot join
- Chronic (more than 2 weeks duration) diarrhea > grade 1
- Prior receipt of naxitamab
- Untreated central nervous system (CNS) metastatic disease
- Pregnant or currently breast feeding: Pregnant participants are excluded from this study because protocol therapy has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the parent with protocol therapy, participants planning to continue breastfeeding are excluded from the study.
- Clinically significant arrhythmias, i.e. those that cause clinical symptoms or require medications for control of symptoms
- Prior allergic reaction to irinotecan or temozolomide
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- Discontinuation of prior irinotecan or temozolomide due to unacceptable toxicity
- Discontinuation of prior GD2 directed immunotherapy due to unacceptable toxicity other than allergic reaction
- Serious intercurrent illness
- Active uncontrolled infection
- Existing major organ dysfunction CTCAE >Grade 2, except for hearing loss and hematological status, kidney, and liver function as described in eligibility criteria
- Concomitant Medication Restrictions:
- Patients may not be receiving immunosuppressive medications including pharmacologic doses of glucocorticoids or immunomodulatory agents due to concern for inhibition of antibody effect. Local and inhaled steroid agents are permitted.
- Patients may not be receiving concurrent anti-cancer agents or radiotherapy.
- Patients may not have received valproic acid within 14 days prior to enrollment.
- Patients may not have received strong CYP3A4 inducers, strong CYP3A4 inhibitors, or strong UGT1A1 inhibitors within 14 days prior to enrollment.
- Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Steven DuBois, MD, MS, 617-632-5460, Steven_Dubois@dfci.harvard.edu
Steven DuBois, MD, MS
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115
Steven DuBois, MD, MS, 617-632-5460, steven_dubois@dfci.harvard.edu
Steven DuBois, MD, MS
About this study
This research is being done to investigate a treatment regimen of Irinotecan, Temozolomide, and Sargramostin, and an immunotherapy called Naxitamab and whether giving Naxitamab more slowly reduces the side effects for participants with relapsed or refractory neuroblastoma.
The name of the study drugs involved in this study are:
- Naxitamab (A type of monoclonal antibody)
- Irinotecan (A standard of care chemotherapy)
- Temozolomide (A standard of care chemotherapy)
- Sargramostim (A standard of care, granulocyte-macrophage colony stimulating factor)
Read the detailed description
This phase 1, single treatment arm, prospective trial is designed to identify the maximally safe infusion duration of Naxitamab in combination with Irinotecan, Sargramostim, and Temozolomide for participants with relapsed, refractory, progressive neuroblastoma. Study doctors are studying different Naxitamab infusion duration levels (how quickly the drug is given into the vein) to determine if this makes a difference in side effects such as pain or infusion reactions.
Not every participant in this research study will receive the same infusion duration of the study drug, Naxitamab. The infusion duration received will depend on the number of participants who have been enrolled in the study and how well an infusion duration is tolerated.
The duration groups will be as follows:
- Duration Level -1: Naxitamab infusion duration will be 5 hours
- Duration Level 1: Naxitamab infusion duration will be 4 hours
- Duration Level 2: Naxitamab infusion duration will be 3 hours
The U.S. Food and Drug Administration (FDA) has approved Naxitamab for patients aged greater than 1 year with relapsed/refractory neuroblastoma in the bone or bone marrow.
The research study procedures include screening for eligibility, in-clinic visits, questionnaires, blood tests, Computerized Tomography (CT) scans, Magnetic Resonance Imaging (MRI) scans, or Meta-Iodine odobenzylguanidine (MIBG) scans, echocardiograms (ECGs), bone marrow aspiration/biopsies.
It is expected that about 18 people will take part in this research study.
YmAbs Therapeutics is supporting this research study by providing naxitamab for the trial and support to measure naxitamab blood levels.
- Being tested
- Naxitamab, Irinotecan, Temozolomide, Sargramostim granulocyte-macrophage colony stimulating factor
- Keywords
- Neuroblastoma, Neuroblastoma Recurrent, Relapsed Neuroblastoma, Refractory Neuroblastoma, Progressive Neuroblastoma
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NaxitamabDrug
Recombinant humanized anti-GD2 monoclonal antibody, single-use vial, via intravenous (into the vein) infusion per protocol.
IrinotecanDrug
Topoisomerase Inhibitor, single-dose vial, via intravenous infusion per standard of care
TemozolomideDrug
Also called Temodal, Temodar
Alkylating agent, capsule, via orally (by mouth) per standard of care
Sargramostim granulocyte-macrophage colony stimulating factorDrug
Also called Leukine
Recombinant human granulocyte-macrophage colony stimulating factor (rhu GM-CSF), multi-use vial, via subcutaneously (under the skin) injection per standard of care
| Study group | Role | What they receive |
|---|---|---|
| Naxitamab Infusion+ Ironotecan + Temozolomide + Sargramostim | Experimental | Participants will be enrolled in a modified 3+3 design to establish a maximum tolerated, infusion duration for Naxitamab. Dosage will start at Duration Level 1 and will de-escalate to Duration Level -1 or escalate to Duration Level 2 according to dose-limiting toxicity (DLT) criteria.
* Baseline visit
* Cycle 1 (21 day cycle):
* Days 1, 3, and 5: Predetermined dose of Naxitamab 1x daily
* Days 1 through 5: Predetermined dose of Temozolomide 1x daily
* Days 1 through 5: Predetermined dose of Ironotecan 1x daily
* Days 6 through 12: predetermined dose of Sargramostim 1x daily
* Cycles 2 through 6 (21 day cycles):
* Imaging on Cycles 2, 4, and 6
* Days 1, 3, and 5: Predetermined dose of Naxitamab 1x daily
* Days 1 through 5: Predetermined dose of Temozolomide 1x daily
* Days 1 through 5: Predetermined dose of Ironotecan 1x daily
* Days 6 through 12: predetermined dose of Sargramostim 1x daily
* End of treatment visit
* Follow up at 1 year post-treatment Receives: Drug: Naxitamab; Drug: Irinotecan; Drug: Temozolomide; Drug: Sargramostim granulocyte-macrophage colony stimulating factor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recommended duration of naxitamab infusion
The duration of naxitamab infusion that is safe and tolerable will be measured using the endpoint of an occurrence of any dose limiting toxicity. A modified 3+3 design will be used, where the 'modification' permits a single duration level de-escalation in addition to the typical duration group level escalation.
Measured: Assessed at the end of the first cycle of treatment, where 1 cycle is 21 days
Secondary outcomes (9)
Frequency of severe infusion related pain as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE)
Frequency of CTCAE severe infusion related pain is defined as the count of all participants that experience a grade 3 or higher pain event as measured by CTCAE version 5.
Measured: Assessed days 1 and 8 for Cycles 1 through 6, where 1 cycle is 21 days
Frequency of severe infusion related pain as measured by the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PEDS PRO-CTCAE)
Frequency of PEDS PRO-CTCAE severe infusion related pain is defined as the count of all participants that experience a score of 3 or greater on Question 1b of the PRO-CTCAE instrument.
Measured: Assessed days 1 and 8 for Cycles 1 through 6, where 1 cycle is 21 days
Frequency of grade 3 or higher infusion related pain as measured by The Face, Legs, Activity, Cry, and Consolability (FLACC) Scale
Frequency of FLACC severe infusion related pain is defined as the count of all participants that experience a score of 7 or greater on the FLACC scale instrument.
Measured: Assessed on every naxitamab infusion day (days 1, 3, and 5) for both Cycle 1 and 2, where 1 cycle is 21 days
Frequency of grade 3 or higher infusion related pain as measured by the Wong-Baker FACES scale
Frequency of FACES severe infusion related pain is defined as the count of all participants that experience a score of 4 or greater on the Wong-Baker FACES instrument.
Measured: Assessed on every naxitamab infusion day (days 1, 3, and 5) for both Cycle 1 and 2, where 1 cycle is 21 days
Frequency of grade 3 or higher related pain as measured by the Numeric Rating Scale (NRS)
Frequency of NRS severe infusion related pain is defined as the count of all participants that experience a score of 7 or greater on the Numeric Rating Scale (NRS) instrument.
Measured: Assessed on every naxitamab infusion day (days 1, 3, and 5) for both Cycle 1 and 2, where 1 cycle is 21 days
Frequency of additional infusion-related toxicity
Frequency of additional infusion-related toxicity will be defined as the count of toxicities which necessitate additional supportive care or interventions (i.e. an outpatient that requires hospital admission; an inpatient that requires ICU transfer).
Measured: Assessed at each clinic visit throughout the duration of treatment and for 35 days following the last dose of treatment, approximately 161 days
Overall Best Response
Overall best response (as defined per International Neuroblastoma Response Committee criteria) at or before the end of 6 cycles of protocol chemoimmunotherapy.
Measured: Assessed at the end of 6 cycles of protocol chemoimmunotherapy, approximately 126 days
Progression-Free Survival (PFS)
PFS time, defined as time from first response until first occurrence of relapse, progression, or death due to disease. Participants without an event will be censored at the time of last contact.
Measured: Assessed quarterly until withdrawal of consent or completion of the study (1 year follow-up after last treatment dose given to last enrolled patient).
Overall Survival (OS)
OS time, defined as the time from first study treatment to death due to any cause, or censored at date of last contact.
Measured: Assessed quarterly until withdrawal of consent or completion of the study (1 year follow-up after last treatment dose given to last enrolled patient).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2025
- Study start27 Jun 2025
- Primary completion1 Jul 2027 (estimated)
- Study completion1 Dec 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.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Participants are enrolled sequentially into cohorts corresponding to different naxitamab infusion durations and following a modified 3+3 design for safety assessment.
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Steven DuBois, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Y-mAbs Therapeutics
- Responsible party
- Sponsor-investigator, Steven DuBois, MD, Dana-Farber Cancer Institute
- Investigators
- Steven DuBois, MD, MS, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07027748
- Sponsor's protocol ID
- 25-211
- Status last verified
- Jul 2026
- First submitted
- 11 Jun 2025
- Last updated
- 14 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: \[contact information for Sponsor Investigator or designee\]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT07027748
- Acronym / sponsor ID
- 25-211
- Keywords
- Neuroblastoma, Neuroblastoma Recurrent, Relapsed Neuroblastoma, Refractory Neuroblastoma, Progressive Neuroblastoma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- Steven DuBois, MDUniversity, hospital or other
- Collaborators
- Y-mAbs Therapeutics
- Study start
- 27 Jun 2025 (actual)
- Primary completion
- 1 Jul 2027 (estimated)
- Study completion
- 1 Dec 2027 (estimated)
- First posted
- 18 Jun 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 research is being done to investigate a treatment regimen of Irinotecan, Temozolomide, and Sargramostin, and an immunotherapy called Naxitamab and whether giving Naxitamab more slowly reduces the side effects for participants with relapsed or refractory neuroblastoma.
The name of the study drugs involved in this study are:
- Naxitamab (A type of monoclonal antibody)
- Irinotecan (A standard of care chemotherapy)
- Temozolomide (A standard of care chemotherapy)
- Sargramostim (A standard of care, granulocyte-macrophage colony stimulating factor)
Detailed description
This phase 1, single treatment arm, prospective trial is designed to identify the maximally safe infusion duration of Naxitamab in combination with Irinotecan, Sargramostim, and Temozolomide for participants with relapsed, refractory, progressive neuroblastoma. Study doctors are studying different Naxitamab infusion duration levels (how quickly the drug is given into the vein) to determine if this makes a difference in side effects such as pain or infusion reactions.
Not every participant in this research study will receive the same infusion duration of the study drug, Naxitamab. The infusion duration received will depend on the number of participants who have been enrolled in the study and how well an infusion duration is tolerated.
The duration groups will be as follows:
- Duration Level -1: Naxitamab infusion duration will be 5 hours
- Duration Level 1: Naxitamab infusion duration will be 4 hours
- Duration Level 2: Naxitamab infusion duration will be 3 hours
The U.S. Food and Drug Administration (FDA) has approved Naxitamab for patients aged greater than 1 year with relapsed/refractory neuroblastoma in the bone or bone marrow.
The research study procedures include screening for eligibility, in-clinic visits, questionnaires, blood tests, Computerized Tomography (CT) scans, Magnetic Resonance Imaging (MRI) scans, or Meta-Iodine odobenzylguanidine (MIBG) scans, echocardiograms (ECGs), bone marrow aspiration/biopsies.
It is expected that about 18 people will take part in this research study.
YmAbs Therapeutics is supporting this research study by providing naxitamab for the trial and support to measure naxitamab blood levels.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Participants are enrolled sequentially into cohorts corresponding to different naxitamab infusion durations and following a modified 3+3 design for safety assessment.
| Arm | Type | Receives |
|---|---|---|
| Naxitamab Infusion+ Ironotecan + Temozolomide + Sargramostim | Experimental | Drug: Naxitamab, Drug: Irinotecan, Drug: Temozolomide, Drug: Sargramostim granulocyte-macrophage colony stimulating factorParticipants will be enrolled in a modified 3+3 design to establish a maximum tolerated, infusion duration for Naxitamab. Dosage will start at Duration Level 1 and will de-escalate to Duration Level -1 or escalate to Duration Level 2 according to dose-limiting toxicity (DLT) criteria. * Baseline visit * Cycle 1 (21 day cycle): * Days 1, 3, and 5: Predetermined dose of Naxitamab 1x daily * Days 1 through 5: Predetermined dose of Temozolomide 1x daily * Days 1 through 5: Predetermined dose of Ironotecan 1x daily * Days 6 through 12: predetermined dose of Sargramostim 1x daily * Cycles 2 through 6 (21 day cycles): * Imaging on Cycles 2, 4, and 6 * Days 1, 3, and 5: Predetermined dose of Naxitamab 1x daily * Days 1 through 5: Predetermined dose of Temozolomide 1x daily * Days 1 through 5: Predetermined dose of Ironotecan 1x daily * Days 6 through 12: predetermined dose of Sargramostim 1x daily * End of treatment visit * Follow up at 1 year post-treatment |
Who may qualify
- Ages
- 1 year to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (56 listed)
- Histologic Diagnosis: Patients must have had histologic verification of neuroblastoma or ganglioneuroblastoma or demonstration of neuroblastoma cells in the bone marrow with elevated urinary catecholamines [i.e. > 2 x upper limit of normal (ULN)], at the time of initial diagnosis.
- Relapsed or Refractory Disease Patients must have ONE of the following:
- Documentation of Disease: Patients must have at least ONE of the following at the time of enrollment:
- Prior Therapy: Prior lines of anticancer therapy allowed as described in eligibility section above by disease status. Washout periods from prior therapy are as follows:
- Age: Patients 1 - 30 years of age at the time of enrollment are eligible for this study.
Key exclusion criteria (17 listed)
- Chronic (more than 2 weeks duration) diarrhea > grade 1
- Prior receipt of naxitamab
- Untreated central nervous system (CNS) metastatic disease
- Pregnant or currently breast feeding: Pregnant participants are excluded from this study because protocol therapy has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the parent with protocol therapy, participants planning to continue breastfeeding are excluded from the study.
- Clinically significant arrhythmias, i.e. those that cause clinical symptoms or require medications for control of symptoms
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 |
|---|---|---|
| NaxitamabRecombinant humanized anti-GD2 monoclonal antibody, single-use vial, via intravenous (into the vein) infusion per protocol. | Drug | Naxitamab Infusion+ Ironotecan + Temozolomide + Sargramostim |
| IrinotecanTopoisomerase Inhibitor, single-dose vial, via intravenous infusion per standard of care | Drug | Naxitamab Infusion+ Ironotecan + Temozolomide + Sargramostim |
| TemozolomideAlso: Temodal, TemodarAlkylating agent, capsule, via orally (by mouth) per standard of care | Drug | Naxitamab Infusion+ Ironotecan + Temozolomide + Sargramostim |
| Sargramostim granulocyte-macrophage colony stimulating factorAlso: LeukineRecombinant human granulocyte-macrophage colony stimulating factor (rhu GM-CSF), multi-use vial, via subcutaneously (under the skin) injection per standard of care | Drug | Naxitamab Infusion+ Ironotecan + Temozolomide + Sargramostim |
Primary outcome
Recommended duration of naxitamab infusion
Time frame: Assessed at the end of the first cycle of treatment, where 1 cycle is 21 days
The duration of naxitamab infusion that is safe and tolerable will be measured using the endpoint of an occurrence of any dose limiting toxicity. A modified 3+3 design will be used, where the 'modification' permits a single duration level de-escalation in addition to the typical duration group level escalation.
9 secondary outcomes
Frequency of severe infusion related pain as measured by NCI Common Terminology Criteria for Adverse Events (CTCAE)
Time frame: Assessed days 1 and 8 for Cycles 1 through 6, where 1 cycle is 21 days
Frequency of severe infusion related pain as measured by the Pediatric Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PEDS PRO-CTCAE)
Time frame: Assessed days 1 and 8 for Cycles 1 through 6, where 1 cycle is 21 days
Frequency of grade 3 or higher infusion related pain as measured by The Face, Legs, Activity, Cry, and Consolability (FLACC) Scale
Time frame: Assessed on every naxitamab infusion day (days 1, 3, and 5) for both Cycle 1 and 2, where 1 cycle is 21 days
Frequency of grade 3 or higher infusion related pain as measured by the Wong-Baker FACES scale
Time frame: Assessed on every naxitamab infusion day (days 1, 3, and 5) for both Cycle 1 and 2, where 1 cycle is 21 days
Frequency of grade 3 or higher related pain as measured by the Numeric Rating Scale (NRS)
Time frame: Assessed on every naxitamab infusion day (days 1, 3, and 5) for both Cycle 1 and 2, where 1 cycle is 21 days
Frequency of additional infusion-related toxicity
Time frame: Assessed at each clinic visit throughout the duration of treatment and for 35 days following the last dose of treatment, approximately 161 days
Overall Best Response
Time frame: Assessed at the end of 6 cycles of protocol chemoimmunotherapy, approximately 126 days
Progression-Free Survival (PFS)
Time frame: Assessed quarterly until withdrawal of consent or completion of the study (1 year follow-up after last treatment dose given to last enrolled patient).
Overall Survival (OS)
Time frame: Assessed quarterly until withdrawal of consent or completion of the study (1 year follow-up after last treatment dose given to last enrolled patient).
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 14 Jul 2026.
- Individual participant dataSharing planned: Yes. The Dana-Farber / Harvard Cancer Center encourages and supports the responsible and ethical sharing of data from clinical trials. De-identified participant data from the final research dataset used in the published manuscript may only be shared under the terms of a Data Use Agreement. Requests may be directed to: [contact information for Sponsor Investigator or designee]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
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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.
Steven DuBois, MD, MS principal investigator
Dana-Farber Cancer Institute
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Naxitamab, Irinotecan and Temozolomide in people with Neuroblastoma and Neuroblastoma Recurrent, run by Steven DuBois, MD at 2 sites 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 56 inclusion and 17 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 14 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 2 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 Steven DuBois, MD, MS (617-632-5460, Steven_Dubois@dfci.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recommended duration of naxitamab infusion (time frame: Assessed at the end of the first cycle of treatment, where 1 cycle is 21 days). 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 14 Jul 2026; AzTrials last refreshed its copy on 8 Nov 2026.