Nivolumab for recurrent or metastatic olfactory neuroblastoma

A Phase II Clinical Trial Evaluating the Efficacy and Safety of Nivolumab Monotherapy for Incurable Recurrent or Metastatic Olfactory Neuroblastoma.

This trial will try nivolumab, a PD‑1 immunotherapy, in adults with recurrent or metastatic olfactory neuroblastoma to see if it controls tumor growth and is safe.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 14 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: National Cancer Center Hospital East (Academic / other)
• Drugs / interventions: chemotherapy, radiation, nivolumab
• Locations: 2 sites (Kashiwa, Chiba and 1 other locations)
• Trial ID: NCT07100704 on ClinicalTrials.gov

What this trial studies

This open‑label Phase 2 trial gives nivolumab alone to adults with recurrent or metastatic olfactory neuroblastoma who are not candidates for curative surgery or radiotherapy. Patients receive either 240 mg on Days 1 and 15 of each 28‑day cycle or 480 mg on Day 1 of each 28‑day cycle, with the dosing schedule chosen by the participant and their treating physician, and treatment continues until disease progression. Key eligibility includes histologic confirmation of olfactory neuroblastoma, prior progression after chemotherapy, at least one measurable lesion per RECIST v1.1, ECOG performance status 0–1, and adequate organ function. The trial will record tumor response and safety/tolerability outcomes during treatment.

Who should consider this trial

Good fit: Adults (≥18 years) with histologically confirmed recurrent or metastatic olfactory neuroblastoma who have progressed after prior chemotherapy, are not eligible for curative local therapy, have measurable disease, ECOG 0–1, and adequate organ function.

Not a fit: Patients with poor performance status, uncontrolled comorbidities, active autoimmune disease, or those who remain eligible for curative local therapy are less likely to benefit from this treatment.

Why it matters

Potential benefit: If effective, nivolumab could shrink tumors or slow disease progression and provide a systemic option for patients who cannot receive curative local therapy.

How similar studies have performed: There are limited and mixed data on PD‑1 inhibitors in this rare tumor type, with only small case reports and series showing occasional responses rather than clear, consistent benefit.

Eligibility criteria

Inclusion Criteria:

1. Written informed consent obtained.
2. Age ≥ 18 years.
3. Histologically confirmed olfactory neuroblastoma.
4. Not eligible for curative local therapy (surgery/radiation).
5. Histological confirmation from recurrent/metastatic lesion or PET-CT evidence.
6. Disease progression after prior chemotherapy.
7. ECOG Performance Status 0-1.
8. Expected survival ≥ 3 months.
9. At least one measurable lesion per RECIST v1.1.
10. Adequate organ function; (1) Absolute Neutrophil Count ≥ 1,000/mm³ (2) Hemoglobin ≥ 8.0 g/dL (3) Platelets ≥ 75,000/mm³ (4) Total bilirubin ≤ 1.5×ULN (≤3.0×ULN for constitutional hyperbilirubinemia) (5) AST/ALT ≤ 3×ULN (≤5×ULN with liver metastasis) (6) Serum creatinine ≤ 1.5×ULN or creatinine clearance ≥ 40 mL/min
11. If the participant is female, she agrees to use contraception and refrain from breastfeeding during the treatment and for 5 months after the treatment. If the participant is male, he agrees to use contraception during the treatment and for 7 months after the treatment.

Exclusion Criteria:

1. Active progressive multiple primary cancers (synchronous multiple cancers and metachronous multiple cancers with a disease-free interval of 5 years or less. However, lesions equivalent to carcinoma in situ or mucosal cancer that are considered curable by local treatment are not included as multiple primary cancers. Additionally, this may not apply if the attending physician determines that early-stage cancer will not be a prognostic factor.).
2. Has a systemic infection that requires treatment.
3. It has been determined that one is infected with HIV or AIDS-related diseases.
4. Having an active autoimmune disease that required systemic therapy.
5. Having interstitial lung disease.
6. Pregnant or breastfeeding.
7. Any other cases where the attending physician determines that the treatment in this protocol is inappropriate.

Where this trial is running

Kashiwa, Chiba and 1 other locations

• National Cancer Center Hospital East — Kashiwa, Chiba, Japan (Recruiting)
• Jichi Medical University Hospital — Shimotsuke, Tochigi, Japan (Recruiting)

Study contacts

• Study coordinator: Yuta Hoshi, MD

Conditions Recurrent/ Metastatic Olfactory Neuroblastoma; Nivolumab