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Phase 2 Study for Relapsed or Refractory Neuroblastoma

This study is for children and young adults (up to age 30) with neuroblastoma that has come back (relapsed) or didn't fully respond to previous treatment (refractory). It's testing if adding a drug called N-803 to a standard treatment combination (irinotecan, temozolomide, hu14.18K322A, and GM-CSF) is safe and effective. The study aims to see if this new combination improves how well patients respond to treatment. The study is currently unclear about its recruitment status and plans to enroll 54 participants. This treatment uses immunotherapy, which helps your body's immune system fight cancer.

Study design
This is a Phase 2 study with a safety run-in, enrolling 54 participants. After an initial safety check, participants will be randomly assigned to receive either the standard treatment alone or the standard treatment plus N-803.
What's involved
Each treatment cycle lasts 21 days, and participants may receive up to 10 cycles. The study involves receiving several medications through IV (into a vein) and subcutaneous (under the skin) injections on specific days within each cycle.
Compensation
Not stated in the trial record.
Follow-up
The study measures treatment response at the end of the first 21-day cycle and again after up to 10 cycles of treatment.

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NCT07085338

A Phase II Study With a Safety Run-In of the Addition of N-803 to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma

Recruiting
PHASE2Up to 30InterventionalTreatment
St. Jude Children's Research Hospital
~54 participants
Updated 2026-08-07 on ClinicalTrials.gov
What's tested:TemozolomideIrinotecanhu14.18K322AN803Sargramostim

At a glance

Recruiting sites
2 of 5 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To evaluate if the administration of N-803 in combination with irinotecan, temozolomide, hu14-18K322A, and GM-CSF in patients with relapsed/refractory neuroblastoma is feasible
Measured over Complete Cycle 1 treatment (each cycle is 21 days)
+2 more outcomes measured

Conditions

Neuroblastoma Recurrent

Explore related trials

Neuroblastoma

Where it's being run

5 sites across 5 states
California1
Colorado1
Michigan1
Pennsylvania1
Tennessee1

Study leadership

  • Sara Federico, MD · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital

Who to contact

Sara Federico, MD
[email protected]
888-226-4343
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Patients must have high-risk neuroblastoma according to COG risk classification at the time of study registration. Patients whose disease was initially considered low or intermediate risk but were then reclassified as high-risk neuroblastoma prior to enrollment are also eligible.
Recurrent/progressive disease after the diagnosis of high-risk neuroblastoma at any time prior to enrollment regardless of response to frontline therapy. (Note that this excludes patients initially considered low or intermediate risk that progressed to high-risk disease but have not progressed after the diagnosis of high-risk neuroblastoma).
Refractory disease: A best overall response of no response/stable disease since diagnosis of high-risk neuroblastoma AND after at least 4 cycles of induction therapy.
Persistent disease: A best overall response of partial response since diagnosis of high-risk neuroblastoma AND after at least 4 cycles of induction therapy
For MIBG non-avid tumors, patients must have at least an FDG-PET avid site and meet the following criteria:
SIZE: Lesion can be accurately measured in at least one dimension with a longest diameter ≥ 10 mm, or for discrete lymph nodes ≥ 15mm on short axis. Lesions meeting size criteria will be considered measurable.
In addition to size, a lesion needs to meet ONE of the following criteria except for patients with parenchymal CNS lesions which will only need to meet size criteria:
If a patient has 3 or more MIBG avid soft tissue lesions, then no biopsy is required.
If a patient has only 1 or 2 MIBG avid soft tissue lesion sites) then biopsy confirmation of neuroblastoma and/or ganglioneuroblastoma in at least one MIBG avid site present at the time of enrollment is required. Soft tissue lesions may be biopsied at any time point prior to enrollment.
The use of dexamethasone as an antiemetic is not permitted.
Irinotecan is a substrate for CYP3A4 (major) and CYP2B6 (major). Patients who have received drugs that are strong inducers or inhibitors of CYP3A4 within 7 days prior to study enrollment are not eligible. The use of strong inducers or inhibitors of CYP3A4 (Appendix II) and CYP2B6 (e.g., carbamazepine) should be avoided for the duration of protocol therapy. Consult drug information references for further information. In addition, concomitant use of BCRP inhibitors (cyclosporine, eltrombopag, gefitinib), and UGT1A1 inhibitors (diclofenac, ketoconazole, probenecid, silibinin, nilotinib, and atazanavir) should be avoided due to potential increased risk of irinotecan toxicity.
Moderate inducers or inhibitors of CYP3A4 (Appendix II) should also be avoided during protocol therapy if reasonable alternatives exist.

What this trial measures

  • To evaluate if the administration of N-803 in combination with irinotecan, temozolomide, hu14-18K322A, and GM-CSF in patients with relapsed/refractory neuroblastoma is feasibleComplete Cycle 1 treatment (each cycle is 21 days)

    Feasibility Measures: Patients evaluable for toxicity in the safety run-in must receive one dose of N-803. The proportion of evaluable patients who successfully complete the protocol-defined treatment regimen in the Safety Run-in phase will be calculated. The reasons for treatment discontinuation or deviations from the protocol will be summarized.

  • To determine if the response rate of N-803 with irinotecan, temozolomide, hu14.18K322A and GM-CSF in patients with relapsed/refractory neuroblastoma is superior to the combination of irinotecan, temozolomide, hu14.18K322A, and GM-CSFComplete Cycle 1 treatment (each cycle is 21 days)

    Tolerability Assessments: Tolerability of the protocol-defined treatment in the Safety Run-in phase will be determined by assessing adverse events and their severity. Adverse events will be categorized based on standard CTCAE v5.0 criteria. Dose modifications or interruptions resulting from treatment-related adverse events will be analyzed.

  • To determine if the response rate of N-803 with irinotecan, temozolomide, hu14.18K322A and GM-CSF in patients with relapsed/refractory neuroblastoma is superior to the combination of irinotecan, temozolomide, hu14.18K322A, and GM-CSFUp to 10 cycles of irinotecan/temozolomide/hu14.18K322A/GM-CSF with or without N-803 in the Phase 2 (each cycle is 21 days)

    Tumor response will be assessed using standard NANT criteria. The primary endpoint for response analysis will be Best Overall Response (BOR), defined as the best response observed prior to progression, cross over or start of another therapy. Point estimates for the response rate (proportion of patients who have BOR of PR or better) will be calculated, together with exact 95% confidence intervals.