Summary
Nivolumab has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. ClinicalTrials.gov lists 8 registered trials linking Nivolumab to Cancer: 1 is currently recruiting; 1 is active or not yet recruiting; 2 have completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT02538666). It plans or enrolled 907 participants. Registration activity spans 2009 to 2025. The literature layer holds 14 publications for this pair: 5 Cochrane reviews, 1 meta-analysis, 3 randomised controlled trial publications and 5 clinical trial publications. The dated items are from 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for Cancer ranks 1226 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Spontaneous regression is rare (<0.5% of cases); 5-year survival without treatment: <5% for advanced NSCLC; ~20% for stage I-II with surgery.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 68.5 (trials 18.5, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 1 recruiting, 1 active / not yet recruiting, 2 completed, 4 other |
| Linked publications | 14 (5 Cochrane reviews, 5 clinical trial publications, 3 randomised controlled trial publications, 1 meta-analysis) |
| Agent type | Biologic |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT02538666 | An Investigational Immuno-therapy Study of Nivolumab, or Nivolumab in Combination With Ipilimumab, or Placebo in Patients With Extensive-Stage Disease Small Cell Lung Cancer (ED-SCLC) After Completion of Platinum-based Chemotherapy 2015 | completed | Phase 3 | 907 |
| NCT06715241 | A MULTICENTER, SEEKING SIGNAL, RANDOMISED, OPEN-LABEL PHASE II OF RELATLIMAB AND NIVOLUMAB VS NIVOLUMAB ALONE IN LOCALLY ADVANCED CERVICAL CANCERS 2024 | active, not recruiting | Phase 2 | 77 |
| NCT03631641 | Nivolumab in Preventing Colon Adenomas in Participants With Lynch Syndrome and a History of Partial Colectomy 2018 | terminated | Phase 2 | 3 |
| NCT03991130 | High Dose IL-2 in Combination With Anti-PD-1 to Overcome Anti-PD-1 Resistance in Metastatic Melanoma and Renal Cell Carcinoma 2019 | terminated | Phase 2 | 6 |
| NCT01024231 | Dose-escalation Study of Combination BMS-936558 (MDX-1106) and Ipilimumab in Subjects With Unresectable Stage III or Stage IV Malignant Melanoma 2009 | completed | Phase 1 | 127 |
| NCT07100405 | TACE Combined With Anti-PD-1 Antibody in Patients With Advanced Hepatocellular Carcinoma: Study on Efficacy and Immune Microenvironment 2025 | recruiting | Not applicable | 50 |
| NCT03371381 | An Efficacy and Safety Study of JNJ-64041757, a Live Attenuated Listeria Monocytogenes Immunotherapy, in Combination With Nivolumab Versus Nivolumab Monotherapy in Participants With Advanced Adenocarcinoma of the Lung 2018 | terminated | PHASE1, PHASE2 | 12 |
| NCT03167177 | QUILT-3.046: NANT Melanoma Vaccine: Combination Immunotherapy in Subjects With Melanoma Who Have Progressed On or After Chemotherapy and PD-1/PD-L1 Therapy 2017 | withdrawn | PHASE1, PHASE2 | — |
Published literature
- Clinical trial publication Long-term follow-up from the OMNIVORE trial: response-adaptive nivolumab and ipilimumab in advanced renal cell carcinomaMcKay RR, Serzan M, Xie W et al. · Journal for immunotherapy of cancer · 2026 · PMID 42386345
- Clinical trial publication Rescue by radiotherapy and anti-CTLA4/PD-1 after failure of anti-PD-1 therapy in patients with metastatic NSCLC: the phase II RECLAIM trialUlas EB, Purcell ND, Houda I et al. · Journal for immunotherapy of cancer · 2026 · PMID 42331381
- Clinical trial publication CHIMERA: A Phase II Study of Neoadjuvant Pembrolizumab in Combination With Cisplatin or Carboplatin and Pemetrexed Followed by Surgery and Adjuvant Pembrolizumab in Resectable Pleural MesotheliomaPasello G, Scattolin D, Faccioli E et al. · Clinical lung cancer · 2026 · PMID 42308637
- Clinical trial publication Randomized non-comparative phase II trial of nivolumab plus paclitaxel in subjects with recurrent/metastatic head and neck squamous cell carcinoma unable for cisplatin-based chemotherapy. NIVOTAX TTCC studyDocampo LI, Basté N, Oliva M et al. · European journal of cancer (Oxford, England : 1990) · 2026 · PMID 42107346
- Clinical trial publication A Multi-Institutional Randomized Phase III Trial of Neoadjuvant Chemoimmunotherapy Followed By Surgery Versus Upfront Surgery in Patients With Resectable Clinical Stage II-III Non-Small Cell Lung Cancer: JCOG2317 (NATCH-ICI)Mitome N, Hamada A, Wakabayashi M et al. · Clinical lung cancer · 2026 · PMID 42107198
- Cochrane review Diagnosis and management of immune checkpoint inhibitor-induced dry mouth and salivary gland dysfunction in oncology patients: a systematic reviewLópez-Pintor RM, González-Serrano J, Villa A et al. · Supportive care in cancer : official journal of the Multinational Association of Supportive Care in Cancer · 2026 · PMID 42397548
- Cochrane review Effect of histology on the efficacy of first-line immune checkpoint inhibitors in advanced non-small cell lung cancer: a systematic review and network meta-analysisLi S, He T, Chen J et al. · Frontiers in immunology · 2026 · PMID 42317330
- Cochrane review Neoadjuvant Chemoimmunotherapy in Resectable NSCLC With PD-L1 Expression < 1%: A Systematic Review and Network Meta-Analysis of Randomized Controlled TrialsMamede I, Taylor S, Nohmi RL et al. · Clinical lung cancer · 2026 · PMID 42247797
- Cochrane review First-line immunotherapy for advanced hepatocellular carcinoma: a network meta-analysis of randomized trials with overall and HBV/HCV-stratified efficacy and safetyChen W, Chen B, Hu C et al. · Frontiers in immunology · 2026 · PMID 42220514
- Cochrane review Efficacy and risk analysis of monotherapy and dual immunotherapy in dMMR/MSI-H metastatic colorectal cancer: a meta-analysis based on randomized controlled trialsWang S, Gu R, Lu J et al. · BMC cancer · 2026 · PMID 42168914
- Meta-analysis Efficacy and Safety of Novel-Agent-Based Front-Line Therapy in Older Adults Aged ≥ 60 Years With Classic Hodgkin Lymphoma: A Systematic Review and Meta-AnalysisSwain RN, Singh C, Jandial A et al. · Hematological oncology · 2026 · PMID 42330019
- Randomized controlled trial Safety and efficacy of intratumoural anti-CTLA4 with intravenous anti-PD1Tselikas L, Susini S, Texier M et al. · Nature · 2026 · PMID 42056527
- Randomized controlled trial Gonadal function and fertility after Hodgkin lymphoma treatment with nivolumab-AVD in the phase 2 GHSG NIVAHL trialRobertz AS, Bühnen I, Meissner J et al. · Blood · 2026 · PMID 41926569
- Randomized controlled trial Quemliclustat and chemotherapy with or without zimberelimab in metastatic pancreatic adenocarcinoma: a randomized phase 1 trialWainberg ZA, Manji GA, Bahary N et al. · Nature medicine · 2026 · PMID 41912809
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Nivolumab approved for Cancer?
Nivolumab has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Cancer would be drug repurposing rather than first-in-human development. This does not mean it is approved for Cancer. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Nivolumab in clinical trials for Cancer?
ClinicalTrials.gov lists 8 registered trials linking Nivolumab to Cancer: 1 is currently recruiting; 1 is active or not yet recruiting; 2 have completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 3 (NCT02538666). It plans or enrolled 907 participants. Registration activity spans 2009 to 2025.
What does the evidence show for Nivolumab in Cancer?
Nivolumab has both completed registered trials and synthesis-level publications linked to Cancer. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.