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Biliary Tract CancerPhase 2NCT02829918

Study of Nivolumab in Patients With Advanced Refractory Biliary Tract Cancers

A Phase 2 study testing Nivolumab in people with Biliary Tract Cancer and Biliary Tract Neoplasms, run by H. Lee Moffitt Cancer Center and Research Institute at 3 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This research study is designed to see if a drug called Nivolumab is effective in treating patients with advanced refractory biliary tract cancers. Nivolumab has been approved by the U.S. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Biliary Tract Cancer, Biliary Tract Neoplasms
What is being tested?
Nivolumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
3 sitesUnited States
Study dates
5 Oct 2016to 24 May 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Must have histologically or cytologically documented carcinoma primary to the intra- or extra-hepatic biliary system or gall bladder with clinical and/or radiologic evidence of unresectable, locally advanced or metastatic disease. Patients with ampullary carcinoma are not eligible.
  • Must have failed or are intolerant to one line of systemic treatment but no more than 2 prior lines of systemic chemotherapy for advanced BTC. Patients who received adjuvant chemotherapy and had evidence of disease recurrence within 6 months of completion of the adjuvant treatment are also eligible. If the patient received adjuvant treatment and had disease recurrence after 6 months, patients will only be eligible after failing or having intolerance to one line of systemic chemotherapy used to treat the disease recurrence.
  • Age ≥ 18
  • Eastern Cooperative Oncology Group (ECOG) Performance Status Assessment of 0 or 1.
  • Must have radiographic measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.
  • Life expectancy of at least 12 weeks (3 months).
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  • For patients who have received prior radiation, cryotherapy, radiofrequency ablation, therasphere, ethanol injection, transarterial chemoembolization (TACE) or photodynamic therapy, the following criteria must be met: 28 days have elapsed since that therapy; Lesions that have not been treated with local therapy must be present and measureable.
  • Must be able to understand and be willing to sign the written informed consent form. Must be willing and able to comply with scheduled visits, treatment schedule, laboratory testing, and other study requirements.
  • All acute toxic effects of any prior treatment have resolved to NCI-CTCAE v4.0 Grade 1 or less at the time of signing the Informed Consent Form (ICF) except for alopecia.
  • Adequate bone marrow, liver and liver function.
  • Women of childbearing potential must have a negative serum or urine pregnancy test (minimum sensitivity of 25 IU/L or equivalent units of HCG) performed within24 hours prior to the start of nivolumab Post-menopausal women (defined as no menses for at least 1 year) and surgically sterilized women are not required to undergo a pregnancy test.
  • Men and women of childbearing potential must agree to use adequate contraception beginning at the signing of the ICF until at least 3 months after the last dose of study drug.
  • Patients with history of hepatitis B and hepatitis C will be eligible but patients with hepatitis B must be started on antiviral therapy prior to beginning study therapy.
  • Availability of archival tumor tissue for biomarkers analysis (FFPE block or cell block will be required).

Who cannot join

  • Active central nervous system (CNS). If CNS metastases are treated and potential participants are at neurologic baseline for at least 2 weeks prior to enrollment, they will be eligible but will need a Brain MRI prior to enrollment. Must be off corticosteroids or on a dose of less than 10 mg per day.
  • Active, known or suspected autoimmune disease. Potential participants with vitiligo, type I diabetes mellitus, residual hypothyroidism due to autoimmune thyroiditis only requiring hormone replacement, or conditions not expected to recur in the absence of an external trigger are permitted to enroll.
  • A condition requiring systemic treatment with either corticosteroids (>10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days of enrollment. Inhaled or topical steroids, and adrenal replacement steroid doses > 10 mg daily prednisone equivalent, are permitted in the absence of active autoimmune disease.
  • Prior therapy with anti-PD-1, anti-PD-L1, anti-PD-L2, anti-CD137, or anti-CTLA-4 antibody (including ipilimumab or any other antibody or drug specifically targeting T-cell costimulation or checkpoint pathways).
  • Previous or concurrent cancer within 3 years prior to treatment start EXCEPT for curatively treated cervical cancer in situ, non-melanoma skin cancer, superficial bladder tumors [Ta (non-invasive tumor), Tis (carcinoma in situ) and T1 (tumor invades lamina propria)].
  • Known history of human immunodeficiency virus (HIV) infection or acquired immunodeficiency syndrome (AIDS).
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  • Child Pugh B or C disease.
  • History of severe hypersensitivity reactions to other monoclonal antibodies.
  • History of allergy or intolerance to study drug components or Polysorbate-80-containing Infusions.
  • Substance abuse, medical, psychological or social conditions that may interfere with the patient's participation in the study or evaluation of the study results.
  • History or concurrent condition of interstitial lung disease of any grade or severely impaired pulmonary function.
  • Unresolved toxicity higher than CTCAE grade1 attributed to any prior therapy/procedure excluding alopecia.
  • Pregnant or breast-feeding patients. Women of childbearing potential must have a negative serum or urine pregnancy test (minimum sensitivity of 25 IU/L or equivalent units of HCG) performed within 24 hours prior to the start of nivolumab and a negative results must be documented before start of treatment.
  • Any illness or medical conditions that are unstable or could jeopardize the safety of the patient and his/her compliance in the study.
  • Excluded Therapies and Medications for Cancer: 1) Anticancer chemotherapy during the study or within 4 weeks of study enrollment. Must have recovered from the toxic effects of the previous anti-cancer chemotherapy (with the exception of alopecia). Anti-cancer therapy is defined as any agent or combination of agents with clinically proven anti-tumor activity administered by any route with the purpose of affecting the malignancy, either directly or indirectly, including palliative and therapeutic endpoints. 2) Hormonal therapy during the study or within 2 weeks of first study enrollment. 3) Radiotherapy to target lesions during study or within 2 weeks of enrollment. 4) An irradiated lesion is considered evaluable only if it has shown enlargement since the completion of last radiation. 5) Bone marrow transplant or stem cell rescue. 6) Investigational drug therapy outside of this trial during or within 4 weeks of first study treatment.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Emory University Winship Cancer Institute

    Atlanta, Georgia, 30322

  • City of Hope Cancer Center

    Duarte, California, 91010

  • H. Lee Moffitt Cancer Center and Research Institute

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: A Phase II Investigator Sponsored Study of Nivolumab in Patients With Advanced Refractory Biliary Tract Cancers

This research study is designed to see if a drug called Nivolumab is effective in treating patients with advanced refractory biliary tract cancers. Nivolumab has been approved by the U.S. Food and Drug Administration (FDA) for treatment of certain types of cancer but is not approved by the FDA for treatment of your type of cancer.

Being tested
Nivolumab
Keywords
advanced biliary tract cancer, refractory biliary tract cancer, digestive diseases, immunotherapy
Medical subject headings
Biliary Tract Neoplasms, Digestive System Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NivolumabDrug

    Also called OPDIVO

    Participants will receive nivolumab at a dose of 240 mg intravenously (IV) every 2 weeks (Q 2W) for 16 weeks (16W) and then 480 every 4 weeks (Q4W) from 17 weeks to end of study.

Study groups in NCT02829918
Study groupRoleWhat they receive
Nivolumab TreatmentExperimentalThis is a single arm study with two stage design using nivolumab in advanced biliary tract cancer (BTC), for participants who have failed or are intolerant to at least one line of therapy and no more than 2 lines of therapy. In the first stage, 18 participants will be accrued. If there is at least one response (or several participants with stable disease based on the study team's discretion), an additional 34 patients will be accrued for a total of 52 patients.
Receives: Drug: Nivolumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall Response Rate (ORR) After 4 Cycles of Treatment

    Response in participants with advanced biliary tract cancers receiving nivolumab as a single agent. ORR is defined as complete responses (CR) plus partial responses (PR) as measured by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.

    Measured: 16 weeks

Secondary outcomes (2)

  1. Overall Survival (OS)

    OS is defined as the time from enrollment to the date of death.

    Measured: Up to 36 months

  2. Progression Free Survival (PFS)

    PFS is defined as the time from first treatment to the date of the first documented tumor progression as determined by the investigator (per RECIST 1.1), or death due to any cause.

    Measured: Up to 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jul 2016 (estimated)
  2. Study start5 Oct 2016
  3. Primary completion27 Mar 2019
  4. Study completion24 May 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
54 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02829918
Sponsor's protocol ID
MCC-18684
Status last verified
Feb 2026
First submitted
8 Jul 2016
Last updated
11 Mar 2026
Data monitoring committee
No

Publications

  • Kim RD, Chung V, Alese OB, El-Rayes BF, Li D, Al-Toubah TE, Schell MJ, Zhou JM, Mahipal A, Kim BH, Kim DW. A Phase 2 Multi-institutional Study of Nivolumab for Patients With Advanced Refractory Biliary Tract Cancer. JAMA Oncol. 2020 Jun 1;6(6):888-894. doi: 10.1001/jamaoncol.2020.0930.

    PubMed 32352498

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Nivolumab in people with Biliary Tract Cancer and Biliary Tract Neoplasms, run by H. Lee Moffitt Cancer Center and Research Institute at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (ORR) After 4 Cycles of Treatment (time frame: 16 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02829918, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Mar 2026.

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