Actively Recruiting
A Phase II Study With a Safety Run-In of N-803 Added to Chemoimmunotherapy for Patients With Relapsed or Refractory Neuroblastoma
Led by St. Jude Children's Research Hospital · Updated on 2026-06-04
54
Participants Needed
4
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to study children and young adults with neuroblastoma that either did not fully respond to previous treatment or has returned after treatment. The study evaluates whether adding the drug N-803 to a standard chemoimmunotherapy combination can be safely given and if it improves the response rate compared to chemoimmunotherapy alone. It also investigates the side effects and compares how long patients live without disease progression and overall survival between the two treatment groups. Participants will receive treatments in cycles lasting 21 days each. The study starts with a safety run-in phase where the first 6 patients receive chemoimmunotherapy plus N-803 to assess safety and dosing. If tolerated, the trial proceeds to phase 2 where patients are randomly assigned to receive either chemoimmunotherapy alone or with N-803. Those in the chemoimmunotherapy group who experience disease progression after two cycles may switch to the combination including N-803. Treatments include irinotecan and temozolomide given intravenously on days 1 to 5, hu14.18K322A intravenously on days 2 to 5, N-803 by injection on day 6, and GM-CSF injections on days 7 to 13. During the study, participants will be closely monitored through blood tests, imaging, and evaluations to assess response and side effects. Researchers will track treatment feasibility, tolerability, response rates, survival outcomes, and toxicity up to 36 months. Pharmacokinetic and biological studies will also be conducted. The total duration varies depending on response and treatment cycles, with up to 10 cycles planned in phase 2.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be younger than 30 years at enrollment.
- Histologic verification of neuroblastoma or neuroblastoma cells in bone marrow with elevated catecholamines at initial diagnosis.
- High-risk neuroblastoma classification according to COG criteria at study registration.
- Must have recurrent, progressive, refractory, or persistent neuroblastoma after at least 4 cycles of induction therapy.
- Presence of at least one measurable lesion in bone, bone marrow, or soft tissue meeting imaging or biopsy criteria.
- Lansky or Karnofsky performance score of 50 or higher.
- Fully recovered from acute toxic effects of prior therapy.
- Meet hematologic, renal, liver, cardiac, pulmonary, and CNS function requirements.
- Use effective contraception if of reproductive potential and have negative pregnancy test if applicable.
You will not qualify if you...
- Pregnancy, breastfeeding, or unwillingness to use contraception.
- Inability to comply with safety monitoring.
- Disease compromising ability to tolerate therapy.
- Prior allogeneic stem cell or solid organ transplant.
- Patients on hemodialysis.
- Active or uncontrolled infections.
- Known HIV, hepatitis B, or hepatitis C infection.
- Need for pharmacologic systemic corticosteroids during treatment (with some exceptions).
- Use of immunosuppressive medications.
- Recent use of enzyme-inducing anticonvulsants or strong CYP3A4 inducers/inhibitors.
- Diagnosis of myelodysplastic syndrome or malignancies other than neuroblastoma.
- Symptoms of congestive heart failure.
- Greater than Grade 2 diarrhea.
- History of disease progression on prior irinotecan/temozolomide/dinutuximab/GM-CSF therapy.
- History of severe allergic reactions to anti-GD2 antibodies.
- Elevated catecholamines without evidence of disease (not eligible).
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 1 cycle of 21 days for initial safety assessment; additional cycles if dose adjustment is needed
Participants receive cycles of irinotecan, temozolomide, hu14.18K322A, GM-CSF, and N-803 to assess safety and tolerability before proceeding to Phase 2.
1 cycle with multiple visits during Days 1-13
Duration - Up to 10 cycles of 21 days each
Participants are randomized to receive chemoimmunotherapy alone or chemoimmunotherapy plus N-803, with treatment cycles repeated up to 10 times.
Multiple visits per 21-day cycle corresponding to drug administration schedule
Duration - Up to 36 months
Participants are monitored for disease progression, survival, and adverse events after treatment completion.
Periodic visits for assessments during follow-up
Trial Site Locations
Total: 4 locations
1
University of California San Francisco
San Francisco, California, United States, 94158
Not Yet Recruiting
2
Children's Hospital of Colorado
Colorado Springs, Colorado, United States, 80902
Not Yet Recruiting
3
Motts Childrens Hospital
Ann Arbor, Michigan, United States, 48109
Not Yet Recruiting
4
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
S
Sara Federico, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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