Feasibility Study of Prolonged Administration of Naxitamab, Irinotecan, and Temozolomide for Patients With Relapsed or Refractory Neuroblastoma

Recruiting now · Phase 1

Conditions studied: Neuroblastoma, Neuroblastoma Recurrent, Relapsed Neuroblastoma, Refractory Neuroblastoma

In brief

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)

Key facts

Study ID

NCT07027748

Run by

Steven DuBois, MD

People needed

18

Starts

2025-06-27

Expected to finish

2027-12-01

Last updated by the study team

2026-07-14

Who can join

Age: 1 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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:
• 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)

You may not qualify if…

• 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
• 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.

Where it is running

• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Dana-Farber Cancer Institute — Boston, Massachusetts, United States (enrolling)

Full record on ClinicalTrials.gov

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