Trial Outcomes & Findings for Rucaparib in Combination With Nivolumab in Patients With Advanced or Metastatic Biliary Tract Cancer Following Platinum Therapy (NCT NCT03639935)

Last Updated: 2025-06-03

Results Overview

Progressive disease is defined as at least a 20% increase in the sum of the longest diameter (LD) of target lesions (with a minimum absolute increase of 5 mm), taking as reference the smallest sum LD recorded since the treatment started, or the appearance of one or more new lesions. A clinical decision of progression by the site investigator will be based on the subject's overall clinical condition, including performance status, clinical symptoms, and laboratory data.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

4 months

Results posted on

2025-06-03

Participant Flow

One participant was enrolled, but never treated, as they did not meet lab requirements on C1D1

Participant milestones

Participant milestones
Measure	Rucaparib and Nivolumab Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Overall Study STARTED	31
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Rucaparib and Nivolumab Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Overall Study Death	3
Overall Study Adverse Event	1

Baseline Characteristics

Rucaparib in Combination With Nivolumab in Patients With Advanced or Metastatic Biliary Tract Cancer Following Platinum Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	23 Participants n=99 Participants
Age, Categorical >=65 years	8 Participants n=99 Participants
Sex: Female, Male Female	14 Participants n=99 Participants
Sex: Female, Male Male	17 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	2 Participants n=99 Participants
Race (NIH/OMB) Asian	2 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	2 Participants n=99 Participants
Race (NIH/OMB) White	25 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	31 participants n=99 Participants

PRIMARY outcome

Timeframe: 4 months

Outcome measures

Outcome measures
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Proportion of Patients Alive and Without Radiological or Clinical Progression at 4 Months	54.8 percentage of participants Interval 36.0 to 72.7

SECONDARY outcome

Timeframe: up 2 years after starting treatment, average of 4 months

The proportion of patients that display a partial response (PR) or complete response (CR) to treatment. Partial response is defined as at least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. There can be no appearance of new lesions. Complete response 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.

Outcome measures

Outcome measures
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
The Proportion of Patients That Respond to Treatment	2 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Outcome measures

Outcome measures
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Progression Free Survival (PFS) Time as Measured From Treatment Start	4.6 months Interval 3.7 to 6.2

SECONDARY outcome

Timeframe: Up to two years post treatment discontinuation

Outcome measures

Outcome measures
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Progression Free Survival (PFS) Time as Measured From Start of 1st Line Platinum Therapy	9.9 months Interval 8.3 to 11.3

SECONDARY outcome

Timeframe: Up to two years post treatment discontinuation

Outcome measures

Outcome measures
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Overall Survival (OS) Time as Measured From Treatment Start	15.9 months Interval 9.8 to 19.4

SECONDARY outcome

Timeframe: Up to two years post treatment discontinuation

Outcome measures

Outcome measures
Measure	Rucaparib and Nivolumab n=31 Participants Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Overall Survival (OS) Time as Measured From Start of 1st Line Platinum Therapy	21.4 months Interval 14.8 to 25.7

Adverse Events

Rucaparib and Nivolumab

Serious events: 17 serious events

Other events: 28 other events

Deaths: 26 deaths

Serious adverse events

Serious adverse events
Measure	Rucaparib and Nivolumab n=31 participants at risk Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Infections and infestations Upper respiratory infection	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
General disorders Abdominal pain	9.7% 3/31 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Musculoskeletal and connective tissue disorders Muscle weakness right-sided	3.2% 1/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Nervous system disorders Seizure	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Infections and infestations Sepsis	16.1% 5/31 • Number of events 5 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	12.9% 4/31 • Number of events 4 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Skin and subcutaneous tissue disorders Bullous dermatitis	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Gastrointestinal disorders Diarrhea	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Gastrointestinal disorders Obstruction gastric	6.5% 2/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Infections and infestations Febrile neutropenia	6.5% 2/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Renal and urinary disorders Biliary tract infection	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Renal and urinary disorders Acute kidney injury	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Blood and lymphatic system disorders Thromboembolic event	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Cardiac disorders Heart failure	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Vascular disorders Hypotension	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
General disorders Non-cardiac chest pain	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Infections and infestations Infections and infestations - Other, specify	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Blood bilirubin increased	6.5% 2/31 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Surgical and medical procedures Surgical and medical procedures - Other, specify	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Respiratory, thoracic and mediastinal disorders Respiratory failure	3.2% 1/31 • Number of events 1 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.

Other adverse events

Other adverse events
Measure	Rucaparib and Nivolumab n=31 participants at risk Rucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15 Rucaparib: Rucaparib 600 mg PO BID days 1-28 Nivolumab: Nivolumab 240 mg IV days 1 and 15
Investigations Pruritus	6.5% 2/31 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Skin and subcutaneous tissue disorders Rash maculo-papular	6.5% 2/31 • Number of events 11 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Alkaline phosphatase increased	6.5% 2/31 • Number of events 15 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Aspartate aminotransferase increased	6.5% 2/31 • Number of events 24 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Gastrointestinal disorders Bloating	6.5% 2/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Psychiatric disorders Confusion	6.5% 2/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
General disorders Dizziness	6.5% 2/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
General disorders Fatigue	6.5% 2/31 • Number of events 23 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
General disorders Headache	6.5% 2/31 • Number of events 3 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Hyperglycemia	12.9% 4/31 • Number of events 19 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Hypocalcemia	9.7% 3/31 • Number of events 7 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Vascular disorders Hypotension	9.7% 3/31 • Number of events 4 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Lymphocyte count decreased	9.7% 3/31 • Number of events 11 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Gastrointestinal disorders Nausea	6.5% 2/31 • Number of events 12 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Neutrophil count decreased	6.5% 2/31 • Number of events 17 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Eye disorders Photosensitivity	6.5% 2/31 • Number of events 2 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.
Investigations Platelet count decreased	9.7% 3/31 • Number of events 30 • All adverse event data (serious and non-serious) collected from the time of the initial study treatment administration through 30 days after the last dose of study treatment; additionally, any serious adverse event occurring more than 30 days after the last study treatment if considered to be related to the study treatment. Data was collected during a 5 year period. Adverse Events and All-Cause Mortality will be evaluated with secondary endpoints as data is still being collected.

Additional Information

ClinicalTrials.gov Admin

University of Michigan Rogel Cancer Center

Phone: 734-936-9499

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place