What is nivolumab studying for rare tumors?

Nivolumab is being studied to see how well it works against advanced or metastatic rare tumors that express PD-L1. Researchers want to find out if this antibody treatment can help patients whose cancers have not responded to standard therapies. This study is led by Instituto do Cancer do Estado de Se3o Paulo and focuses on a tissue-agnostic approach regardless of tumor origin.

Who may be eligible for this rare tumor clinical trial?

Adults aged 18 years or older with advanced rare cancers that show PD-L1 expression at a CPS of 10 or higher may be eligible. Participants must have progressed after standard treatments and have a good performance status. Both men and women can join if they meet contraceptive or fertility conditions. Please review eligibility criteria for more details.

Is there any cost to participate in this rare tumor clinical trial?

Study-related care, including the study drug and all visits, is provided at no cost. However, personal travel and other expenses may vary and are not guaranteed to be covered by the study.

Actively Recruiting

Phase 2

Age: 18Years +

All Genders

ID06638931

Phase II Study of Nivolumab, an Anti-PD1 Antibody, for Treating Advanced Rare Tumors with PD-L1 Expression

Led by Instituto do Cancer do Estado de São Paulo · Updated on 2026-04-15

Participants Needed

Research Sites

95 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the tissue-agnostic effectiveness of the anti-PD1 antibody nivolumab in patients with advanced or metastatic rare tumors that express PD-L1 with a combined positive score of 10 or higher. This phase II basket trial includes patients whose cancers have progressed despite standard treatments. Rare tumors, defined by low incidence rates, collectively represent a significant portion of cancers with generally poor prognosis and limited clinical trial representation. Participants receive intravenous nivolumab at a dose of 480 mg every 4 weeks, continuing until disease progression, limiting toxicity, or for up to 12 months if responses are stable or improving. After treatment ends or discontinuation, patients are followed by phone every 60 days until death. This innovative approach targets the molecular characteristic PD-L1 regardless of tumor origin, aiming to provide new therapeutic options across many rare cancer types. During the study, researchers assess tumor response using RECIST v1.1 criteria and monitor biomarkers including PD-L1 expression, circulating tumor DNA, and microvesicles to understand treatment effects. Patients undergo regular evaluations to measure objective response and progression-free survival. Safety is closely monitored throughout the treatment and follow-up periods. The study is conducted across multiple centers to overcome challenges in rare cancer research and is expected to last about four years.

CONDITIONS

Brief Title

Agnostic Therapy in Rare Solid Tumors

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Age 18 years or older
PD-L1 expression with a combined positive score (CPS) of 10 or higher
Progression or intolerance to approved treatments for the specific rare tumor
Radiological evidence of disease progression after last routine treatment
ECOG performance status of 0 or 1
Measurable lesion per RECIST v1.1 criteria
Male participants must be infertile, have no fertile partner, or have a fertile partner agreeing to use contraception during and 6 months after treatment, and agree to abstain from sperm donation during this time
Female participants must be infertile or agree to use contraception during and 6 months after treatment
Estimated life expectancy greater than 12 weeks
Preserved organ function including specific blood counts and liver and kidney function
Diagnosis of a rare cancer confirmed by histopathology, including a specified list of rare tumor types

You will not qualify if you...

Previous treatment with immunotherapy (immune checkpoint inhibitors)
Pregnant or breastfeeding
Limiting comorbidities as judged by the investigator
Active infection
Major surgery within the last 4 weeks
Heart failure of functional class II or greater
Myocardial infarction or stroke within the last 6 months
History of pulmonary fibrosis or pneumonitis
Autoimmune diseases except controlled thyroid conditions or vitiligo without immunosuppressors
Second invasive primary tumor diagnosed in last 3 years except treated non-melanoma skin tumors
Prolonged QT interval
Uncontrolled central nervous system metastases; stable treated CNS metastases allowed if no corticosteroids are used
Presence of meningeal carcinomatosis
Worsening kidney or liver function in the last 14 days
History of solid organ transplantation
Untreated acquired immunodeficiency; HIV patients must have undetectable viral load
Chronic corticosteroid use exceeding 10 mg/day prednisone or equivalent except compensated adrenal insufficiency patients

Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Up to 12 months

Participants receive intravenous nivolumab 480 mg every 4 weeks. Treatment continues until limiting toxicity, disease progression, or for a maximum of 12 months (13 cycles) if stable disease or better is maintained.

Approximately 13 visits every 4 weeks (in-person)

Follow-up

Duration - Variable duration after treatment

After treatment ends, participants are followed by the research team via telephone every 60 days until death.

Telephone contact every 60 days