Trial Outcomes & Findings for Study of the Combination of DKN-01 and Nivolumab in Previously Treated Patients With Advanced Biliary Tract Cancer (BTC) (NCT NCT04057365)

Last Updated: 2025-08-13

Results Overview

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

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

up to 1 year

Results posted on

2025-08-13

Participant Flow

Participant milestones

Participant milestones
Measure	DKN 01 and Nivolumab Safety Run in * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth
Overall Study STARTED	15	0
Overall Study COMPLETED	13	0
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Reasons for withdrawal
Measure	DKN 01 and Nivolumab Safety Run in * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth
Overall Study Withdrawal by Subject	2	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	DKN 01 and Nivolumab Safety Run in n=15 Participants * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	Total n=15 Participants Total of all reporting groups
Age, Customized 18 - 65 years	7 Participants n=5 Participants	0 Participants n=7 Participants	7 Participants n=5 Participants
Age, Customized >65 years	8 Participants n=5 Participants	0 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	0 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	0 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants	0 Participants n=7 Participants	15 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 1 year

Population: A Simon 2 stage design will be used, and the study did not meet criteria to proceed beyond the safety run-in cohort.

Outcome measures

Outcome measures
Measure	DKN 01 and Nivolumab Safety Run in n=13 Participants * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth
Overall Response Rate	0 Participants	—

SECONDARY outcome

Timeframe: up to 1 year

Population: A Simon 2 stage design will be used, and the study did not meet criteria to proceed beyond the safety run-in cohort.

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 of at least 5 mm. (Note: the appearance of one or more new lesions is also considered progression).

Outcome measures

Outcome measures
Measure	DKN 01 and Nivolumab Safety Run in n=13 Participants * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth
Progression Free Survival	49 days Interval 40.0 to 96.0	—

SECONDARY outcome

Timeframe: up to 1 year

Population: A Simon 2 stage design will be used, and the study did not meet criteria to proceed beyond the safety run-in cohort.

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

Outcome measures

Outcome measures
Measure	DKN 01 and Nivolumab Safety Run in n=13 Participants * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth
Overall Survival	12 Participants	—

SECONDARY outcome

Timeframe: up to 1 year

Population: The study did not proceed beyond the initial safety run-in cohort.

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.

Outcome measures

Outcome measures
Measure	DKN 01 and Nivolumab Safety Run in n=13 Participants * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth	DKN 01 and Nivolumab * DKN-01 will be administered intravenously on day 1 and day 15 of each 28 day cycle * Nivolumab will be administered intravenously on day 1 and day 15 of each 28 day cycle Nivolumab: Nivolumab is believed to work by attaching to and inhibiting a specific protein in the cancer that controls parts of the immune system (the system in the body that fights off infections and diseases) by shutting down certain immune responses. The investigators believe that nivolumab will inhibit the protein, thus allowing the immune cells to recognize and destroy cancer cells. DKN-01: DKN-01 is believed to work by attaching to and inhibiting (stopping) a specific pathway in the cells that is responsible for processes such as cell growth
Treatment Emergent Adverse Events Alkaline phosphatase increased	1 Participants	—
Treatment Emergent Adverse Events Dyspnea	1 Participants	—
Treatment Emergent Adverse Events Hypokalemia	1 Participants	—
Treatment Emergent Adverse Events Abdominal pain	2 Participants	—
Treatment Emergent Adverse Events Lymphopenia	2 Participants	—
Treatment Emergent Adverse Events Alanine aminotransferase (ALT) increased	1 Participants	—
Treatment Emergent Adverse Events Aspartate aminotransferase (AST) increased	1 Participants	—

Adverse Events

DKN 01 and Nivolumab Safety Run in

Serious events: 0 serious events

Other events: 13 other events

Deaths: 1 deaths

DKN 01 and Nivolumab

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Additional Information

Joseph Franses, MD, PhD

Massachusetts General Hospital

Phone: 617-724-4000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place