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Nivolumab

Phase 2

Melanoma | Small molecule | Oncology |Exelixis, Inc.|Last Updated: Apr 30, 2026

Target and mechanism

Molecular targetPDCD1
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment14

FDA Designations

No designations recorded

Clinical trial landscape

Nivolumab · 4 trials · 7 indications

Phase 2 4
NCT04472767Cabozantinib Combined With Ipilimumab/Nivolumab and TACE in Patients With Hepatocellular CarcinomaHepatocellular Carcinoma
RECRUITING35 Analytics
NCT04091750Nivolumab/Ipilimumab Plus Cabozantinib in Patients With Unresectable Advanced MelanomaMelanoma
ACTIVE NOT_RECRUITING14 Analytics
NCT04197310Cabozantinib and Nivolumab for Carcinoid TumorsCarcinoid Tumor
COMPLETED19 Analytics
NCT03316586A Phase II Study of Nivolumab in Combination With Cabozantinib for Metastatic Triple-negative Breast CancerBreast Cancer
COMPLETED18 Analytics
PHASE2RECRUITING
Cabozantinib Combined With Ipilimumab/Nivolumab and TACE in Patients With Hepatocellular Carcinoma
Hepatocellular CarcinomaUnlock trial analytics
PHASE2ACTIVE NOT_RECRUITING
Nivolumab/Ipilimumab Plus Cabozantinib in Patients With Unresectable Advanced Melanoma
MelanomaUnlock trial analytics
PHASE2COMPLETED
Cabozantinib and Nivolumab for Carcinoid Tumors
Carcinoid TumorUnlock trial analytics
PHASE2COMPLETED
A Phase II Study of Nivolumab in Combination With Cabozantinib for Metastatic Triple-negative Breast Cancer
Breast CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants with Progression-free Survival at 6 Months
6 months

This is defined as the percentage of subjects who are free of progression 6 months after study treatment start. Progression is defined death, radiographic progression or clinical deterioration attributed disease progression as judged by an investigator. Radiographic progression is defined using the modified Response Evaluation Criteria in Solid Tumors Criteria (mRECIST), as a 20% increase in the sum of diameters of of viable (enhancing) target lesions and/or appearance of one or new lesions and/or unequivocal progression of existing non-target lesions.

Complete Response Rate
From date of registration until first date of disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 1 year.

Complete Response (CR) is defined as the disappearance of all target lesions, determined by two separate observations conducted not less than 4 weeks apart. There can be no appearance of new lesions.

The Progression Free Survival (PFS) for Nivolumab/Ipilimumab Plus Cabozantinib in Patients With Unresectable Advanced Melanoma.
1 year

The PFS rate for nivolumab/ipilimumab plus cabozantinib in patients with unresectable advanced melanoma using imRECIST.

Objective Response Rate (ORR)
46 months

ORR is defined by RECIST 1.1 criteria, the percentage of subjects with a confirmed complete response or partial response at any time during treatment. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by MRI or CT: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the diameters of target lesions; Overall Response (OR) = CR + PR.

Overall Response Rate
2 years

Overall response rate (ORR) is defined as the percentage of patients with complete or partial response evaluated by RECIST 1.1. Per RECIST 1.1: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.

Secondary Endpoints

Overall Survival of Patients who Received Cabozantinib with Ipilimumab/Nivolumab and TACE
From date of registration for up to 18 months after last patient is enrolled or until death from any cause, whichever came first.
Percentage of Grade 3-5 Adverse Events
From the start date of treatment until 4 weeks after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 1 year.
Progression Free Survival
From the start date of treatment until 4 weeks after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 1 year.
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cabozantinib with Ipilimumab/Nivolumab and TACEEXPERIMENTALSubjects receive Cabozantinib 40 mg daily on days 1-28 of a 28 day cycle, this is to be started 7-14 days after the last TACE procedure. Nivolumab 480 mg IV on day 1 of a 28 day cycle (cycle 2 and beyond), this is to be started 7-14 days after the last TACE procedure. Nivolumab: 3mg/kg IV on day 1 of a 21 day cycle x 1 dose. Ipilimumab: 1 mg/kg on day 1 of a 21 day cycle x 1 dose TACE: Within 3-4 weeks of cycle 1 day 1; may be done up to 3 times (9-12 weeks total), the intervals between each TACE treatment can vary based on investigator's discretion
Single ArmEXPERIMENTALNivolumab 3mg/kg IV plus Ipilimumab 1mg/kg IV every 3 weeks x 4 cycles with Cabozantinib 40mg PO daily for 12 weeks (Induction); Followed by Maintenance therapy: Nivolumab 480mg IV every 4 weeks for up to 92 weeks; Cabozantinib 40mg PO daily for up to 92 weeks; Maintenance therapy will continue for up to 92 weeks to complete 2 years total of treatment if tolerating therapy well and disease is controlled.
Nivolumab and CabozantinibEXPERIMENTALThe research study procedures include screening for eligibility and study treatment including evaluations and follow up visits. * Cabozantinib will be administered at a dose of 40mg orally, once daily * Nivolumab will be given at a dose of 240mg every 14 days, intravenously Retreat Phase (Optional) * Participants may elect to stop nivolumab and cabozantinib with confirmed CR after at least 24 weeks of treatment. * Participants who elect to stop and then the condition progresses after stopping study treatment may be eligible to resume nivolumab and cabozantinib therapy. * This resumption will be termed as a retreatment second course phase and is available only while the study remains open and the subject meets specified criteria.
Nivolumab + CabozantinibEXPERIMENTAL* Nivolumab was administered every 28 days at a dose of 480mg given intravenously over 30 minutes (+/- 10 minutes) using a volumetric pump with 0.2 to 1.2 micron pore size, low protein binding polyethersulfone membrane in-line filter * Cabozantinib was administered orally, once daily for 28 days at a dose of 40 mg.

Interventions

NameTypeDescription
NivolumabDRUGGiven IV
IpilimumabDRUGGiven IV
CabozantinibDRUGGiven PO
Transarterial ChemoembolizationPROCEDURETACE treatment will be administered using either the DEB-TACE or cTACE modality in a series of up to 3 individual procedures within the 9-12 weeks following Day 21 (= cycle 1 day 21) of a patient's first infusion of nivolumab/ipilimumab. The first TACE treatment should start no more than 7 working days after being cycle 1 day 21.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Histologic or radiographic diagnosis of hepatocellular carcinoma * At least one lesion amenable to TACE treatment * Child-Pugh A-B7 (B7 based on Albumin allowed) * Not a candidate for resection or transplantation * Age ≥ 18 years. * Performance status: ECOG performance status ...

Countries:United States
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Recent Changes (Last 90 Days)

LOWMay 24, 2026NCT04472767studyFirstPostDate: changed
LOWMay 24, 2026NCT04091750studyFirstPostDate: changed

Frequently asked questions about Nivolumab

What is Nivolumab used for?

Nivolumab is an investigational oncology drug being studied in Phase 2 clinical trials for melanoma, breast cancer, carcinoid tumor, and hepatocellular carcinoma. It is a small molecule inhibitor of PDCD1, and is being developed by Exelixis, Inc. (EXEL). It is not FDA approved and remains in clinical development.

What does Nivolumab target?

Nivolumab targets PDCD1, also known as programmed cell death protein 1, and acts as an inhibitor of this target. By inhibiting PDCD1, it is designed to modulate immune responses in cancer. This mechanism is being evaluated in several Phase 2 trials across different tumor types.

Who makes Nivolumab?

Nivolumab is being developed by Exelixis, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker EXEL. The company is conducting Phase 2 clinical trials of Nivolumab in combination with other agents for multiple cancer indications.

What phase is Nivolumab in?

Nivolumab is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. There are multiple Phase 2 trials ongoing or completed, including studies in melanoma, breast cancer, carcinoid tumors, and hepatocellular carcinoma.

What clinical trials is Nivolumab in?

Nivolumab is being studied in four Phase 2 trials: NCT03316586 for metastatic triple-negative breast cancer, NCT04091750 for unresectable advanced melanoma, NCT04197310 for carcinoid tumors, and NCT04472767 for hepatocellular carcinoma. These trials are conducted in the United States and enroll adult patients.

Is Nivolumab the same as any other drug?

Nivolumab is not known by any alternative names. It is a distinct investigational agent being developed by Exelixis, Inc. for use in combination therapies across several cancer types. Its unique identifier is CHEMBL2108738.