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

A Phase II Study With a Safety Run-In of the Addition of N-803, a Novel IL-15 Super-Agonist, to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 54 (estimated)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 30 Years
• Healthy volunteers: Not accepted

Summary

The study participant is being asked to take part in this research study because the participant has been diagnosed with neuroblastoma that did not fully respond to previous treatment (refractory), or it has returned after treatment (relapsed). Primary Aims * 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 and tolerable * 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-CSF Secondary Aims * To describe the toxicity profile of N-803 administered with irinotecan, temozolomide, hu14.18K322A and GM-CSF * To evaluate and compare the progression free survival (PFS) and overall survival (OS) of and between patients receiving irinotecan, temozolomide, hu14.18K322A and GM-CSF with and without N-803

Detailed description

This is a randomized phase 2 study with a safety assessment run-in conducted in children with relapsed or refractory neuroblastoma to evaluate the feasibility, tolerability, and response to chemoimmunotherapy backbone (irinotecan, temozolomide, hu14.18K322A and GM-CSF) with or without N-803. The first 6 patients included in the safety assessment run-in phase will receive irinotecan, temozolomide, hu14.18K322A, GM-CSF and N-803. If fewer than 2 patients in the first cohort of 6 patients experience a DLT in Cycle 1, then the trial will proceed Phase 2. If 2 or more of the first 6 patients experience a DLT in Cycle 1 then the N-803 will be dose reduced, and 6 more patients will be enrolled in the safety assessment run-in. Once the safety assessment run-in phase is completed, patients will be enrolled onto the phase 2 study. In the phase 2 study, patients will be randomized to receive either chemoimmunotherapy alone (Arm A) or chemoimmunotherapy plus N-803 (Arm B). Patients treated on Arm A who experience disease progression may cross over at any time point after completing Cycle 2 and receive therapy administered on Arm B. Pharmacokinetic and correlative biology studies will be performed throughout the study.

Conditions

• Neuroblastoma Recurrent

Interventions

Type	Name	Description
DRUG	Temozolomide	IV, Days 1-5
DRUG	Irinotecan	IV, Days 1-5
DRUG	hu14.18K322A	IV over 4 hours daily times 4 doses, Days 2-5.
DRUG	N803	Subcutaneous (SC), Day 6.
DRUG	Sargramostim	Subcutaneous injection (preferred) or IV, Days 7-13.

Timeline

• Start date: 2025-11-10
• Primary completion: 2028-02-01
• Completion: 2029-02-01
• First posted: 2025-07-25
• Last updated: 2026-02-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study