Nivolumab for Cancer

Recruiting at 60 trial locations

Overseen By Aparna Hegde

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: National Cancer Institute (NCI)

Must not be taking: Anticancer investigational agents

Disqualifiers: Chemotherapy, Radiotherapy, Allogeneic transplant, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 4

Duration: Up to 2 years

300 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effects and safety of nivolumab, a type of immunotherapy, on individuals with certain cancers that have spread or cannot be surgically removed. Nivolumab helps the immune system attack cancer cells and may prevent their growth. This trial suits those with cancers such as melanoma, lung, or bladder cancer, particularly if previous treatments were ineffective or if they have autoimmune disorders. Participants will receive nivolumab through an IV every four weeks, and the study will collect various samples to monitor progress. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications. However, if you have received certain types of immunotherapy, there are specific waiting periods before starting nivolumab. It's best to discuss your current medications with the trial team to get a clear answer.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Research has shown that nivolumab has been tested in various cancer treatments, and important safety information is available. In a large study on nivolumab, researchers found that while many patients tolerated the drug well, some experienced serious immune-related side effects. These occur because the treatment can cause the immune system to attack healthy parts of the body, not just the cancer. Common serious reactions include infections like urinary tract infections and issues with organs such as the kidneys.

Other studies have demonstrated a promising balance of safety and effectiveness over several years. However, rare but serious side effects can occur. Patients considering participation in trials with nivolumab should weigh these potential risks and benefits. It is crucial to discuss any concerns with healthcare providers before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Nivolumab is unique because it uses a mechanism called immune checkpoint inhibition to treat cancer. Unlike traditional chemotherapy that directly attacks cancer cells, nivolumab works by blocking a protein called PD-1 on immune cells, which helps the body's own immune system recognize and destroy cancer cells more effectively. Researchers are excited about this treatment because it has the potential to provide longer-lasting responses and fewer side effects compared to conventional treatments. This approach represents a significant shift towards harnessing the body's natural defenses to fight cancer.

What evidence suggests that nivolumab might be an effective treatment for cancer?

Research has shown that nivolumab works well for some types of cancer. In studies with patients who have non-small cell lung cancer (NSCLC) and kidney cancer, those treated with nivolumab lived longer. For advanced skin cancer (melanoma), long-term results indicated that patients on nivolumab lived longer than those on other treatments. These findings suggest that nivolumab helps the immune system fight cancer cells, slowing their growth and spread. In this trial, participants will receive nivolumab to further evaluate its effectiveness. Although this treatment is still being tested for autoimmune diseases, the positive results from cancer studies suggest it could be effective. ^{6 7 8 9 10}

Who Is on the Research Team?

Hussein A. Tawbi

Principal Investigator

University of Texas MD Anderson Cancer Center LAO

Are You a Good Fit for This Trial?

Adults with advanced or inoperable cancer and an autoimmune disorder (like lupus, rheumatoid arthritis, or multiple sclerosis) can join this trial. They should have a life expectancy over 12 weeks, be relatively active (ECOG 0-2), and have not had certain treatments recently. Those with controlled hepatitis B/C are eligible; however, pregnant women must use contraception.

Inclusion Criteria

My kidney function, measured by creatinine level or GFR, is within the normal range.

Your total bilirubin level is not more than twice the normal limit at the study hospital.

I am HIV-positive, on treatment, and my viral load has been undetectable for the last 6 months.

Exclusion Criteria

I haven't had chemotherapy or radiotherapy in the last 2 weeks.

You are currently taking any other experimental cancer drugs.

I have been treated with drugs targeting PD-1 or PD-L1 before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Patients receive nivolumab intravenously over 30 minutes every 4 weeks for up to 2 years, with regular collection of blood, CSF, tissue, stool, and urine samples

Up to 2 years

Every 4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment completion

100 days

What Are the Treatments Tested in This Trial?

Interventions

• Nivolumab

Trial Overview The trial is testing Nivolumab's effectiveness and safety for patients who have both cancer and autoimmune diseases. It's a phase Ib study to see how well the immune system responds to this monoclonal antibody treatment in attacking cancer cells that spread or cannot be surgically removed.

How Is the Trial Designed?

Group I: Treatment (nivolumab) Experimental Treatment 2 Interventions

Patients receive nivolumab IV over 30 minutes every 4 weeks for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients also undergo collection of blood, CSF, tissue, stool, and urine samples throughout the trial.

Nivolumab is already approved in United States, European Union, Canada, Switzerland for the following indications:

Approved in United States as Opdivo for:

• Advanced or metastatic gastric cancer
• Gastroesophageal junction cancer
• Esophageal adenocarcinoma
• Melanoma
• Non-small cell lung cancer
• Renal cell carcinoma
• Hodgkin lymphoma
• Head and neck squamous cell carcinoma
• Urothelial carcinoma
• Colorectal cancer
• Hepatocellular carcinoma
• Esophageal squamous cell carcinoma

Approved in European Union as Opdivo for:

• Melanoma
• Non-small cell lung cancer
• Renal cell carcinoma
• Hodgkin lymphoma
• Head and neck squamous cell carcinoma
• Urothelial carcinoma
• Colorectal cancer
• Gastric cancer
• Gastroesophageal junction cancer
• Esophageal adenocarcinoma

Approved in Canada as Opdivo for:

• Melanoma
• Non-small cell lung cancer
• Renal cell carcinoma
• Hodgkin lymphoma
• Head and neck squamous cell carcinoma
• Urothelial carcinoma
• Colorectal cancer
• Gastric cancer
• Gastroesophageal junction cancer
• Esophageal adenocarcinoma

Approved in Switzerland as Opdivo for:

• Melanoma
• Non-small cell lung cancer
• Renal cell carcinoma
• Hodgkin lymphoma
• Head and neck squamous cell carcinoma
• Urothelial carcinoma
• Colorectal cancer
• Gastric cancer
• Gastroesophageal junction cancer
• Esophageal adenocarcinoma

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Published Research Related to This Trial

Nivolumab (Opdivo) significantly improves overall survival and response rates in previously-treated patients with advanced nonsquamous non-small cell lung cancer (NSCLC) compared to docetaxel, as shown in the CheckMate 057 trial.

Nivolumab has a manageable adverse event profile and is better tolerated than docetaxel, making it a valuable treatment option for patients who have progressed after chemotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nivolumab: A Review in Advanced Nonsquamous Non-Small Cell Lung Cancer. Keating, GM.[2018]

Nivolumab, an anti-PD-1 antibody, was administered to a 63-year-old female patient with metastatic melanoma after renal transplantation, highlighting the potential for anti-tumor activity in high-risk patients, despite the challenges of concurrent immunosuppression.

The patient experienced acute renal allograft rejection and renal failure shortly after starting nivolumab, indicating significant risks associated with this treatment in transplant recipients, but she later showed a positive clinical response after re-challenging with nivolumab while on hemodialysis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antitumor activity of nivolumab on hemodialysis after renal allograft rejection. Ong, M., Ibrahim, AM., Bourassa-Blanchette, S., et al.[2023]

In a study of 45 patients with metastatic melanoma treated with the combination of nivolumab and ipilimumab, the disease control rate was 54% and the objective response rate was 29%, indicating moderate efficacy in a real-world setting.

The treatment had a high toxicity profile, with 88% of patients experiencing adverse events, and 44% discontinuing treatment due to these events, highlighting the need for careful monitoring and management of side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-world efficacy and toxicity of combined nivolumab and ipilimumab in patients with metastatic melanoma. Parakh, S., Randhawa, M., Nguyen, B., et al.[2019]

Nivolumab and pembrolizumab are FDA-approved monoclonal antibodies that target PD-1, helping to enhance T cell activity against cancer by blocking the PD-1/PD-L1 interaction, which allows cancer cells to escape immune detection.

Both drugs have shown efficacy across multiple cancer types, including melanoma and non-small cell lung cancer, and are considered interchangeable options for treating these conditions, which may help manage the rising costs of cancer therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nivolumab and pembrolizumab: Monoclonal antibodies against programmed cell death-1 (PD-1) that are interchangeable. Prasad, V., Kaestner, V.[2022]

Combining nivolumab and ipilimumab shows improved effectiveness in treating metastatic melanoma compared to using either drug alone, based on a retrospective analysis of approximately 7700 patients.

The combination therapy presents a safety profile that includes immune-related adverse events from both drugs, indicating that while the combination is effective, it also carries a heightened risk of toxicity that needs further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Retrospective Side Effect Profiling of the Metastatic Melanoma Combination Therapy Ipilimumab-Nivolumab Using Adverse Event Data. Soldatos, TG., Dimitrakopoulou-Strauss, A., Larribere, L., et al.[2022]

In a meta-analysis of five studies involving 2399 patients, nivolumab was found to significantly lower the risk of hematological toxicities such as anemia, neutropenia, and leukopenia compared to other treatments.

The relative risks for these conditions were notably low, indicating that nivolumab may be a safer option regarding hematological side effects, which is important for clinicians when considering treatment options.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The risks of hematological toxicities of nivolumab in cancer patients: A PRISMA-compliant meta-analysis. Shi, Z., Liu, X., Chen, M., et al.[2023]

Nivolumab, a PD-1 receptor inhibitor, shows an objective response rate of 18% and a 1-year overall survival rate of 45% in patients with advanced non-small-cell lung cancer, based on a meta-analysis of 20 studies involving 3404 patients.

The presence of PD-L1 expression in patients is associated with a better response to nivolumab, indicating that PD-L1 status can help predict treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The benefit and risk of nivolumab in non-small-cell lung cancer: a single-arm meta-analysis of noncomparative clinical studies and randomized controlled trials. Zhao, B., Zhang, W., Yu, D., et al.[2019]

Nivolumab, an immune checkpoint inhibitor, significantly improves overall survival and progression-free survival in patients with advanced stage squamous cell non-small cell lung cancer (NSCLC) compared to docetaxel, as shown in the CHECKMATE 017 study.

The safety and tolerability of nivolumab are favorable, and interestingly, the expression of the PD-L1 ligand does not predict treatment outcomes, suggesting that other factors may influence the effectiveness of this therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nivolumab in the treatment of metastatic squamous non-small cell lung cancer: a review of the evidence. Lim, JS., Soo, RA.[2018]

Nivolumab, an immune checkpoint inhibitor, significantly improved overall survival and progression-free survival compared to docetaxel in patients with advanced squamous non-small cell lung cancer (NSCLC) in the CheckMate 017 trial.

Nivolumab was better tolerated than docetaxel, with manageable adverse effects, making it a promising treatment option for patients who have already undergone chemotherapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nivolumab: a review in advanced squamous non-small cell lung cancer. Keating, GM.[2022]

In a study of 79 pre-treated patients with advanced non-small cell lung cancer, nivolumab significantly prolonged progression-free survival, especially in patients who had a partial response to prior treatments.

The occurrence of skin rash during nivolumab treatment was associated with improved long-term survival, suggesting that this side effect may indicate a favorable response to the therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Analysis of Pleiotropic Effects of Nivolumab in Pretreated Advanced or Recurrent Non-small Cell Lung Cancer Cases. Akano, Y., Kuribayashi, K., Funaguchi, N., et al.[2020]

Nivolumab, an immune checkpoint inhibitor for metastatic melanoma, can cause immune-related adverse events (irAEs) such as colitis, which were observed in two patients after treatment.

The development of these irAEs is linked to a Th1 dominant immune response, and serum levels of C-reactive protein (CRP) and interleukin-6 (IL-6) may serve as potential biomarkers to monitor and manage these adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Severe colitis after PD-1 blockade with nivolumab in advanced melanoma patients: potential role of Th1-dominant immune response in immune-related adverse events: two case reports. Yoshino, K., Nakayama, T., Ito, A., et al.[2020]

Nivolumab, a monoclonal antibody targeting the programmed death receptor-1, has shown promising efficacy in treating advanced malignant melanoma, with nearly 25% of patients achieving a partial tumor response in a phase II trial involving previously treated stage III/IV patients.

The treatment demonstrated a durable clinical benefit, with a median progression-free survival of 172 days and an acceptable safety profile, as less than 18% of patients experienced severe adverse events.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nivolumab: a review of its use in patients with malignant melanoma. Deeks, ED.[2021]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7699139/

Real-World, Long-Term Outcomes of Nivolumab Therapy ... Studies in lung cancer and renal cell carcinoma indicate that response to ICIs is associated with survival, [15,16] suggesting that overall response may be a ...

2. opdivo.com opdivo.com/clinical-trial-results

Clinical Trial Results Select your cancer type above to see how OPDIVO, alone or in combination with other therapies, performed in clinical trials.

3. opdivohcp.com opdivohcp.com/efficacy

Efficacy Information | OPDIVO® (nivolumab) In NSCLC patients receiving OPDIVO 3 mg/kg every 2 weeks with YERVOY 1 mg/kg every 6 weeks, immune-mediated pneumonitis occurred in 9% (50/576) of patients, ...

4. news.bms.com news.bms.com/news/details/2021/Six-and-a-Half-Year-Outcomes-for-Opdivo-nivolumab-in-Combination-with-Yervoy-ipilimumab-Continue-to-Demonstrate-Durable-Long-Term-Survival-Benefits-in-Patients-with-Advanced-Melanoma/default.aspx

Six-and-a-Half-Year Outcomes for Opdivo (nivolumab) in ... With a minimum follow-up of 6.5 years, median overall survival (OS) was 72.1 months with Opdivo plus Yervoy (95% CI: 38.2-NR), the longest ...

5. ascopubs.org ascopubs.org/doi/10.1200/JCO.22.01503

Five-Year Survival Outcomes With Nivolumab Plus ... We present 5-year results from CheckMate 227 Part 1, in which nivolumab plus ipilimumab improved overall survival (OS) versus chemotherapy ...

6. opdivohcp.com opdivohcp.com/safety

Safety Information | OPDIVO® (nivolumab) The most frequent serious adverse reactions reporting in ≥2% of patients who received OPDIVO with chemotherapy were urinary tract infection (4.9%), acute kidney ...

7. opdivo.com opdivo.com/side-effects

What you should know about side effects OPDIVO and OPDIVO Qvantig can cause your immune system to attack normal organs and tissues in any area of your body and can affect the way they work.

8. accc-cancer.org accc-cancer.org/docs/mediaroom/mediaroom/pdf/opdivo-bms-fact-sheet-3-5-15

Product Fact Sheet for OPDIVO® (nivolumab) The safety of OPDIVO was evaluated in Trial 1, a randomized, open-label trial in which. 370 patients with unresectable or metastatic melanoma received OPDIVO 3 ...

9. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11196337/

Safety of nivolumab monotherapy in five cancer types This is the largest study examining the safety of nivolumab-treated patients in real-world clinical practice including rare but potentially serious TRAEs.

10. frontiersin.org frontiersin.org/journals/immunology/articles/10.3389/fimmu.2025.1605958/full

The real-world safety of Nivolumab: a pharmacovigilance ... Furthermore, this trial demonstrated superior 5-year overall survival rates for combination therapy (52%) and nivolumab monotherapy (44%) ...