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Biliary Tract CancerPhase 2NCT03639935

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

A Phase 2 study testing Rucaparib and Nivolumab in people with Biliary Tract Cancer, run by University of Michigan Rogel Cancer Center 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

Investigators hypothesize that following first-line platinum based chemotherapy, rucaparib in combination with nivolumab, will improve progression-free survival and overall survival in BTC patients. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Biliary Tract Cancer
What is being tested?
Rucaparib and Nivolumab
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
32 participants
Where
3 sitesUnited States
Study dates
28 Mar 2019to 30 Apr 2024
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

  • Patients must have a pathologically confirmed adenocarcinoma of the biliary tract (intra-hepatic, extra-hepatic (hilar, distal) or gall bladder) that is not eligible for curative resection, transplantation, or ablative therapies. Tumors of mixed histology are excluded.
  • Patients must have received 1st line platinum-based systemic chemotherapy for advanced BTC for 4-6 months without radiologic or clinical progression. Last systemic infusion of 1st line platinum-based therapy may not be more than 4 weeks from study informed consent. Prior peri-operative chemotherapy is permitted provided it was completed > 6 months from start of platinum-based therapy for advanced disease.
  • Prior surgical resection, radiation, chemoembolization, radioembolization or other local ablative therapies are permitted if completed > 4 weeks prior to enrollment AND if patient has recovered to < 1 grade 1 toxicity.
  • Patients must have measurable disease (as per RECISTv1.1) in at least one site not previously treated with radiation or liver directed therapy (including bland, chemo- or radio-embolization, or ablation) either within the liver or in a metastatic site unless the patient has had complete response to 1st line platinum-based therapy.
  • Age≥18 years
  • Child-Pugh score of A or B7 (Scoring system used to assess the prognosis of chronic liver disease, mainly cirrhosis)
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  • ECOG performance status of 0-1 (Eastern Cooperative Oncology Group scoring system used to quantify general well-being and activities of daily life; scores range from 0 to 5 where 0 represents perfect health and 5 represents death.)
  • Ability to understand and willingness to sign IRB-approved informed consent
  • Available archived tissue (FFPE block or 20 unstained slides from prior core biopsy or surgery)
  • Must be able to tolerate CT and/or MRI with contrast
  • Adequate organ function obtained ≤ 2 weeks prior to registration

Who cannot join

  • Diagnosis of immunodeficiency, or received systemic steroid therapy, or any other form of immunosuppressive therapy within 14 days prior to trial treatment. Short bursts of steroids of 5-7 days (for COPD exacerbation or other similar indication) are allowed.
  • Prior history of solid organ transplantation or brain metastasis (unless treated and stable)
  • Patients may not have undergone a major surgical procedure < 4 weeks prior to registration
  • Active second malignancy other than non-melanoma skin cancer or cervical carcinoma in situ. Patients with history of malignancy are eligible provided primary treatment of that cancer was completed > 1 year prior to registration and the patient is free of clinical or radiologic evidence of recurrent or progressive malignancy.
  • Ongoing active, uncontrolled infections (afebrile for > 48 hours off antibiotics)
  • Have received a live vaccine within 30 days of planned start of the study therapy
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  • Have a psychiatric illness, other significant medical illness, or social situation which, in the investigator's opinion, would limit compliance or ability to comply with study requirements
  • Pregnant or breastfeeding since rucaparib and/or nivolumab may harm the fetus or child. All females of childbearing potential (not surgically sterilized and between menarche and 1-year post menopause) must have a blood or urine test to rule out pregnancy within 2 weeks prior to registration.
  • Women of child-bearing potential and men must agree to use 2 methods of adequate contraception (hormonal plus barrier or 2 barrier forms) OR abstinence prior to study entry, for the duration of study participation, and for 6 months (for women) and 7 months (for men) following completion of study therapy
  • Participants with an active, known or suspected autoimmune disease which may affect vital organ function, or has/may require systemic immunosuppressive therapy for management. Participants with type I diabetes mellitus, hypothyroidism only requiring hormone replacement, skin disorders (such as vitiligo, psoriasis, or alopecia) not requiring systemic treatment, or conditions not expected to recur in the absence of an external trigger are permitted to enroll.
  • Participants with a condition requiring systemic treatment with either corticosteroids (>10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days of start of study treatment. Inhaled or topical steroids, and adrenal replacement steroid doses >10 mg daily prednisone equivalent, are permitted in the absence of active autoimmune disease.
  • Patients may not have previously received anti PD1/PDL1 antibodies or PARP inhibitor for treatment of this cancer.

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Rogel Cancer Center

    Ann Arbor, Michigan, 48109

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

Other trials in United States

About this study

Official title: Phase II Multi-Center Study of PARP Inhibitor Rucaparib in Combination With Anti-PD-1 Antibody Nivolumab in Patients With Advanced or Metastatic Biliary Tract Cancer Following Platinum Therapy

Investigators hypothesize that following first-line platinum based chemotherapy, rucaparib in combination with nivolumab, will improve progression-free survival and overall survival in BTC patients.

Being tested
Rucaparib, Nivolumab
Keywords
Metastatic, Platinum, Rucaparib, Nivolumab, PARP Inhibitor, Anti-PD-1 Antibody
Medical subject headings
Biliary Tract Neoplasms, Neoplasm Metastasis

What participants receive

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

  • RucaparibDrug

    Rucaparib 600 mg PO BID days 1-28

  • NivolumabDrug

    Nivolumab 240 mg IV days 1 and 15

Study groups in NCT03639935
Study groupRoleWhat they receive
Rucaparib and NivolumabExperimentalRucaparib 600 mg PO BID days 1-28 Nivolumab 240 mg IV days 1 and 15
Receives: Drug: Rucaparib; Drug: Nivolumab

What is being measured

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

  1. Proportion of Patients Alive and Without Radiological or Clinical Progression at 4 Months

    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.

    Measured: 4 months

Secondary outcomes (5)

  1. The Proportion of Patients That Respond to Treatment

    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.

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

  2. Progression Free Survival (PFS) Time as Measured From Treatment Start

    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.

    Measured: Up to 2 years

  3. Progression Free Survival (PFS) Time as Measured From Start of 1st Line Platinum Therapy

    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.

    Measured: Up to two years post treatment discontinuation

  4. Overall Survival (OS) Time as Measured From Treatment Start

    Measured: Up to two years post treatment discontinuation

  5. Overall Survival (OS) Time as Measured From Start of 1st Line Platinum Therapy

    Measured: Up to two years post treatment discontinuation

Timeline

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

  1. First posted21 Aug 2018
  2. Study start28 Mar 2019
  3. Primary completion7 Feb 2023
  4. Study completion30 Apr 2024

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
32 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03639935
Sponsor's protocol ID
UMCC 2018.044
Other IDs
HUM00142974
Status last verified
May 2025
First submitted
17 Aug 2018
Last updated
3 Jun 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: IPD that underlie the results published in peer reviewed research articles, after deidentification.

Publications

  • Mohan A, Griffith KA, Goff LW, Crysler O, Enzler T, Cardin DB, Gunchick V, Tsang AP, Dippman D, Young AS, Olivei AC, Mannan R, Rao A, Frankel TL, Kumar-Sinha C, Zalupski MM, Sahai V. A phase 2 multicenter trial of rucaparib and nivolumab as maintenance therapy in patients with advanced biliary tract cancer: BilT-02. Clin Cancer Res. 2026 Jul 23. doi: 10.1158/1078-0432.CCR-25-1114. Online ahead of print.

    PubMed 42489677

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Rucaparib and Nivolumab in people with Biliary Tract Cancer, run by University of Michigan Rogel Cancer Center 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 11 inclusion and 12 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 3 Jun 2025. 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 21 Aug 2024). 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?

Proportion of Patients Alive and Without Radiological or Clinical Progression at 4 Months (time frame: 4 months). 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 NCT03639935, 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 3 Jun 2025.

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