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NCT04057365

Study of the Combination of DKN-01 and Nivolumab in Previously Treated Patients With Advanced Biliary Tract Cancer (BTC)

Terminated Phase 2 Results posted Last updated 13 August 2025
What this trial tests

Phase 2 trial testing Nivolumab in Biliary Tract Cancer in 15 participants. Terminated before completion.

Timeline
7 October 2019
Primary endpoint
25 September 2022
25 September 2022

Quick facts

Lead sponsorMassachusetts General Hospital
PhasePhase 2
StatusTerminated
Study typeINTERVENTIONAL
Allocationnon randomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment15
Start date7 October 2019
Primary completion25 September 2022
Estimated completion25 September 2022
Sites2 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Massachusetts General Hospital

Who can join

18 and older, any sex, with Biliary Tract Cancer. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Overall Response Rate Primary · up to 1 year

Overall response is evaluated using Response Evaluation Criteria in Solid Tumors criteria (RECIST 1.1). A participant is considered to have responded if they achieve either of the following outcomes: * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm * Partial Response (PR): At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters

GroupValue95% CI
DKN 01 and Nivolumab Safety Run in0
Progression Free Survival Secondary · up to 1 year

Progression-Free Survival (PFS) is defined as the time from registration to the earlier of disease progression or death due to any cause. Participants alive without disease progression are censored at date of last disease evaluation. Disease progression is assessed using RECIST 1.1 criteria: \- Progressive Disease(PD): At least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase o

GroupValue95% CI
DKN 01 and Nivolumab Safety Run in4940 – 96
Overall Survival Secondary · up to 1 year

Overall Survival (OS) is defined as the time from registration to death due to any cause, or censored at date last known alive.

GroupValue95% CI
DKN 01 and Nivolumab Safety Run in12
Treatment Emergent Adverse Events Secondary · up to 1 year

Treatment emergent adverse events (TEAEs) are undesirable events not present prior to study treatment, or an already present event that worsens either in intensity or frequency following the treatment. TEAEs reported below were assessed as grade 3 or higher according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v 5.0) guidelines.

Abdominal pain
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in2
Lymphopenia
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in2
Alanine aminotransferase (ALT) increased
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in1
Aspartate aminotransferase (AST) increased
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in1
Alkaline phosphatase increased
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in1
Dyspnea
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in1
Hypokalemia
GroupValue95% CI
DKN 01 and Nivolumab Safety Run in1

Adverse events — posted to ClinicalTrials.gov

Time frame: up to 1 year. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

DKN 01 and Nivolumab Safety Run in
Serious: 0/13 (0%)
Deaths: 1/13
DKN 01 and Nivolumab
Serious: 0
Deaths: 0
Other adverse events (79 terms — click to expand)

ReactionSystemDKN 01 and Nivolumab Safet…DKN 01 and Nivolumab
Aspartate aminotransferase increasedInvestigations
AnemiaBlood and lymphatic system disorders
DyspneaRespiratory, thoracic and mediastinal disorders
HyponatremiaMetabolism and nutrition disorders
Alanine aminotransferase increasedInvestigations
FatigueGeneral disorders
Abdominal painGastrointestinal disorders
Alkaline phosphatase increasedInvestigations
AnorexiaGastrointestinal disorders
AscitesGastrointestinal disorders
DiarrheaGastrointestinal disorders
Lymphocyte count decreasedInvestigations
NauseaGastrointestinal disorders
Blood bilirubin increasedInvestigations
FeverGeneral disorders
HypoalbuminemiaMetabolism and nutrition disorders
Peripheral sensory neuropathyNervous system disorders
Platelet count decreasedInvestigations
PruritusSkin and subcutaneous tissue disorders
Edema limbsGeneral disorders
Generalized muscle weaknessMusculoskeletal and connective tissue disorders
ArthralgiaMusculoskeletal and connective tissue disorders
Back painMusculoskeletal and connective tissue disorders
ChillsGeneral disorders
ConstipationGastrointestinal disorders
CoughRespiratory, thoracic and mediastinal disorders
DehydrationMetabolism and nutrition disorders
DizzinessNervous system disorders
HeadacheNervous system disorders
HyperglycemiaMetabolism and nutrition disorders
HypophosphatemiaMetabolism and nutrition disorders
HypoxiaRespiratory, thoracic and mediastinal disorders
Neutrophil count decreasedInvestigations
PainGeneral disorders
Rash maculo-papularSkin and subcutaneous tissue disorders
Sinus tachycardiaCardiac disorders
Lung infectionInfections and infestations
Abdominal distensionGastrointestinal disorders
ArthritisMusculoskeletal and connective tissue disorders
AspirationRespiratory, thoracic and mediastinal disorders

Data from ClinicalTrials.gov NCT04057365 adverse events section.

Sponsor's own description

This research is studying the effect of the combination of how two study drugs (Nivolumab and DKN-01) works in people with advanced biliary tract cancer.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Wnt/β-catenin signaling in cancers and targeted therapies.
    Yu F, Yu C, Li F, Zuo Y, et al · · 2021 · cited 565× · PMID 34456337 · DOI 10.1038/s41392-021-00701-5
  2. PD-L1, TMB, MSI, and Other Predictors of Response to Immune Checkpoint Inhibitors in Biliary Tract Cancer.
    Rizzo A, Ricci AD, Brandi G. · · 2021 · cited 207× · PMID 33535621 · DOI 10.3390/cancers13030558
  3. Characteristics of the cancer stem cell niche and therapeutic strategies.
    Ju F, Atyah MM, Horstmann N, Gul S, et al · · 2022 · cited 84× · PMID 35659296 · DOI 10.1186/s13287-022-02904-1
  4. The evolving roles of Wnt signaling in stem cell proliferation and differentiation, the development of human diseases, and therapeutic opportunities.
    Yu M, Qin K, Fan J, Zhao G, et al · · 2024 · cited 81× · PMID 38292186 · DOI 10.1016/j.gendis.2023.04.042
  5. Signaling pathways in the regulation of cancer stem cells and associated targeted therapy.
    Manni W, Min W. · · 2022 · cited 52× · PMID 36226253 · DOI 10.1002/mco2.176
  6. Treatment of Intrahepatic Cholangiocarcinoma-A Multidisciplinary Approach.
    Krenzien F, Nevermann N, Krombholz A, Benzing C, et al · · 2022 · cited 49× · PMID 35053523 · DOI 10.3390/cancers14020362
  7. Current and novel therapeutic opportunities for systemic therapy in biliary cancer.
    Marin JJG, Prete MG, Lamarca A, Tavolari S, et al · · 2020 · cited 45× · PMID 32694694 · DOI 10.1038/s41416-020-0987-3
  8. Wnt/β-Catenin Signaling and Resistance to Immune Checkpoint Inhibitors: From Non-Small-Cell Lung Cancer to Other Cancers.
    Muto S, Enta A, Maruya Y, Inomata S, et al · · 2023 · cited 44× · PMID 36672698 · DOI 10.3390/biomedicines11010190

Verify or expand the search:

Other trials of Nivolumab

Trials testing the same drug.

Other recruiting trials for Biliary Tract Cancer

Currently open trials in the same condition.

Other Massachusetts General Hospital trials

Trials by the same sponsor.

Verify against primary sources

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Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04057365.

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