Biliary Track CancerNCT07853690
Histotripsy in Biliary Tract Cancer
A study testing Gemcitabine, Cisplatin and Durvalumab in people with Biliary Track Cancer, run by NYU Langone Health at 1 site in United States.
Trial summary
The purpose of this pilot study is to examine the safety and feasibility of histotripsy in two cohorts of 12 patients each with Biliary Track Cancer (BTC), with a plan to expand to a larger multi-center study if both safety and feasibility are demonstrated. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Biliary Track Cancer
- What is being tested?
- Gemcitabine, Cisplatin and Durvalumab
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 5 Oct 2026to Sep 2028
- Phase
- Interventional
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
- Both Cohorts
- Age >/= 18 at time of screening
- Willing and able to provide informed consent for the trial
- Histologically or cytologically confirmed biliary tract cancer (BTC) including cancers of the intrahepatic bile ducts, extrahepatic bile ducts, and gallbladder
- Locally advanced or metastatic disease not eligible for resection or transplant
- RECIST v 1.1 measurable disease
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- No prior systemic therapy for the treatment of locally advanced/metastatic disease a. Recurrent disease after surgical resection must have occurred >/= 6 months from completion of adjuvant therapy
- Eastern Cooperative Oncology Group Performance Status (ECOG PS) 0-2
- Adequate organ and bone marrow function, defined as:
- Hemoglobin > 9.0 g/dL
- Absolute neutrophil count > 1.5 x 109/L
- Platelet count > 100 x 109/L
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) < 2.5 x ULN
- Creatinine within normal limits OR measured creatinine clearance (CL) >50 mL/min OR calculated creatinine clearance (CL) >50 mL/min as determined by Cockcroft-Gault
- Willing and able to undergo general anesthesia and histotripsy procedure
- A tumor(s) for histotripsy treatment ≤3 cm in longest diameter
- Note: a maximum of three (3) tumors are allowed to be treated with histotripsy during the index procedure, regardless of how many tumors the subject has
- Subject has an adequate acoustic window to visualize targeted tumor(s) using ultrasound imaging
- Cohort 1 1) Additional organ function as below:
- Bilirubin < 1.5 x upper limit of normal (ULN). i. Note: patients with Gilbert's Syndrome may be allowed in consultation with the treating physician and the study PI Cohort 2
- Additional organ function as below:
- Bilirubin > 1.5x ULN due to BTC-related biliary tract obstruction after at least 1 attempt at percutaneous/endoscopic stenting
- With the exception of hyperbilirubinemia, no contraindication to chemoimmunotherapy
Who cannot join
- Both Cohorts
- Prior exposure to immune-mediated therapy, including, but not limited to, anti-CTLA-4, anti-PD-1, anti-PD-L1 and anti-PD-L2 antibodies
- History of allogeneic or solid organ transplant
- Active or prior documented autoimmune disorders. The following are exceptions to this criterion:
- Patients with vitiligo or alopecia.
- Patients with hypothyroidism (eg, following Hashimoto syndrome) stable on hormone replacement.
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- Any chronic skin condition that does not require systemic therapy.
- Patients with celiac disease controlled by diet alone.
- Patients without active disease in the last 5 years may be included but only after consultation with the study PI.
- Uncontrolled intercurrent illness, including, but not limited to:
- Ongoing or active infection including but not limited to the below. Patients on prophylactic antibiotics may be eligible after discussion with the study PI.
- Tuberculosis (tuberculosis testing by local institutional practice is required prior to receipt of chemoimmunotherapy) ii. Hepatitis B (HCV) defined as presence of hepatitis B surface antigen [HBsAg] and/or anti-HBcAb with detectable hepatitis B virus [HBV] DNA ≥10 IU/mL (testing is required prior to receipt of chemoimmunotherapy).
- 1. Patients with a past or resolved HBV infection (defined as the presence of hepatitis B core antibody [anti-HBc] and the absence of HBsAg) are eligible.
- Hepatitis C (HCV) defined as positive HCV antibody (testing is required prior to receipt of chemoimmunotherapy).
- 1. Patients positive for hepatitis C (HCV) antibody are eligible only if the polymerase chain reaction is negative for HCV RNA.
- Uncontrolled HIV/AIDS. Testing for HIV 1/2 antibodies is required prior to receipt of chemoimmunotherapy. Patients with known HIV are allowed on study provided they have:
- A stable regimen of highly active anti-retroviral therapy (HAART)
- No requirement for concurrent antibiotics or antifungal agents for the prevention of opportunistic infection
- A CD4 count > 250 cells/mcL
- An undetectable HIV viral load on standard PCR-based testing b. Symptomatic congestive heart failure, uncontrolled hypertension, unstable angina pectoris, MI/TIA/CVA within 3 months prior to study enrollment, uncontrolled cardiac arrhythmia.
- Active interstitial lung disease (ILD) or history of immune-mediated pneumonitis.
- Active primary immunodeficiency. e. Psychiatric illness/social situations that would limit compliance with study requirement, substantially increase the risk of incurring AEs, or compromise the ability of the patient to give written informed consent.
- 5) Other co-morbid disease or condition that would cause undue risk and/or interfere with the conduct or interpretation of the trial in the opinion of the treating investigator 6) Patients with a prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessments of the investigational regimen. Patients whose prior or concurrent malignancy natural history and/or treatment does NOT have the potential to impact safety or study assessments are eligible.
- 7) Any unresolved toxicity NCI Common Terminology Criteria for Adverse Event (CTCAE) Grade ≥2 from a previous anticancer therapy, with the exception of alopecia, vitiligo, and the laboratory values defined in the inclusion criteria. Patients with irreversible toxicity not reasonably expected to be exacerbated by treatment may be included after consultation with the study PI.
- 8) Histotripsy-related criteria:
- Targeted tumor(s) treatment volume overlaps a non-targeted tumor visible via imaging
- Targeted tumor(s) is not clearly visible with diagnostic ultrasound and computed tomography (CT) or magnetic resonance (MR) imaging
- Targeted tumor(s) is located in liver segment 1
- The Planned Treatment Volume intended to cover the targeted tumor includes or encompasses any portion of the main portal vein, common hepatic duct, common bile duct, gallbladder or stomach/bowel.
- 9) Known allergy or hypersensitivity to any of the study drugs or any of the study drug excipients.
- 10) Subject is pregnant or planning to become pregnant or nursing (lactating). 11) Subjects of reproductive potential who are not willing to use effective birth control from screening to 180 days after the last dose of GCD/P or 90 days after last dose of D/P monotherapy.
- 12) Subject is enrolled in another investigational trial and/or is taking investigational medication and/or has been treated with an investigational device </= 30 days prior to planned procedure date.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
NYU Langone Health
New York, New York, 10016
About this study
Official title: A Pilot Study Assessing the Safety and Efficacy of Histotripsy as an Adjunctive Treatment for Biliary Tract Cancer
The purpose of this pilot study is to examine the safety and feasibility of histotripsy in two cohorts of 12 patients each with Biliary Track Cancer (BTC), with a plan to expand to a larger multi-center study if both safety and feasibility are demonstrated. All patients from cohort 1 will be evaluated for objective response rate (ORR), duration of response (DOR), disease control rate (DCR), progression free survival (PFS) and overall survival (OS) of histotripsy in combination with chemoimmunotherapy. Toxicity profile of histotripsy will be characterized for this cohort.
For Cohort 2, in addition to the above evaluations, changes in serum bilirubin levels (total and direct) over time, time to biliary decompression or normalization of bilirubin (total and direct), and percent of patients rendered eligible for chemoimmunotherapy with GCD/P will also be assessed.
Read the detailed description
For cohort 1, histotripsy will be added to chemoimmunotherapy regime with gemcitabine + cisplatin + durvalumab/pembrolizumab (GCD/P) in patients with untreated locally advanced/metastatic BTCs. Peripheral blood sampling and imaging assessment will be done for all patients at baseline and after every 4 treatment cycles.
For cohort 2, histotripsy will be added in the setting of biliary tract obstruction in patients with untreated locally advanced/metastatic BTC and concomitant biliary tract obstruction unrelieved by biliary stenting. Patients who become eligible for chemoimmunotherapy will undergo treatment with GCD/P as per Cohort 1 until disease progression or intolerable toxicity following the same peripheral blood and imaging schedule as Cohort 1.
- Conditions
- Biliary Track Cancer
- Being tested
- Gemcitabine, Cisplatin, Durvalumab, Pembrolizumab, Histotripsy
- Keywords
- BTC
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GemcitabineDrug
1000 mg/m2 over 30 minutes on Days 1, 8, of each cycle (every 3 weeks), infused according to local labeling.
CisplatinDrug
The reconstituted solution should be administered by IV infusion over a 6- to 8-hour period. Cisplatin is given on Days 1 and 8 of each cycle (every 3 weeks), infused according to local labeling
DurvalumabDrug
Given on day 1 of each cycle (every 3 weeks), infused according to local labeling. Administer infusion solution intravenously over 60 minutes through an intravenous line containing a sterile, low-protein binding 0.2 or 0.22 micron in-line filter.
PembrolizumabDrug
Given on day 1 of each cycle (every 3 weeks), infused according to local labeling. Administer diluted solution intravenously over 30 minutes through an intravenous line containing a sterile, non-pyrogenic, low-protein binding 0.2 micron to 5 micron in-line or add-on filter.
HistotripsyDevice
Histotripsy is a non-invasive medical technique that uses focused ultrasound waves to mechanically break down and destroy targeted tissues through a process called cavitation.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Patients eligible for chemoimmunotherapy | Experimental | Eligible patients will undergo histotripsy and therapy with GCD/P (gemcitabine 1,000 mg/m2 IV and cisplatin 25 mg/m2 IV days 1 and 8 with durvalumab 1,500 mg IV or pembrolizumab 200 mg IV day 1 in 21-day cycles) until disease progression or intolerable toxicity. Patients will have the option of narrowing to maintenance durvalumab/pembrolizumab with or without gemcitabine after 4-6 months of GCD/P as per standard-of-care. Receives: Drug: Gemcitabine; Drug: Cisplatin; Drug: Durvalumab; Drug: Pembrolizumab; Device: Histotripsy |
| Cohort 2: Patients not eligible for chemoimmunotherapy | Experimental | Eligible patients will undergo histotripsy (with or without standard of care biliary tract stenting) and then be monitored with weekly hepatic function panels and clinic visits to monitor status of biliary tract obstruction and for candidacy for chemoimmunotherapy. Patients who become eligible for chemoimmunotherapy will undergo treatment with GCD/P as per Cohort 1 until disease progression or intolerable toxicity following the same peripheral blood and imaging schedule as Cohort 1. Receives: Drug: Gemcitabine; Drug: Cisplatin; Drug: Durvalumab; Drug: Pembrolizumab; Device: Histotripsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate (ORR)
Objective response rate (ORR) defined as the proportion of patients with a best overall response of either Complete Response (CR) or Partial Response (PR), as determined by standardized imaging criteria (RECIST v1.1)
Measured: up to 24 months
Incidence of serious adverse events (SAEs)
Incidence of serious adverse events (SAEs) defined as events that are life-threatening, require hospitalization, result in disability, etc.
Measured: up to 24 months
Incidence of major procedure-related complications through 30 days post-histotripsy
Incidence of major procedure-related complications through 30 days post-histotripsy defined as an AE classified as CTCAE grade 3 or higher
Measured: through 30 days post-histotripsy
Secondary outcomes (11)
Duration of response (DOR)
Duration of response (DOR) defined as the time from the first documentation of a Complete Response (CR) or Partial Response (PR) until the date of disease progression or death from any cause
Measured: up to 24 months
Disease control rate (DCR)
Disease control rate (DCR) defined as the proportion of patients who achieve a best overall response of Complete Response (CR), Partial Response (PR), or Stable Disease (SD)
Measured: up to 24 months
Progression free survival (PFS)
Progression free survival (PFS) defined as the time from the date of first treatment to the earliest date of documented disease progression (as determined by imaging per RECIST v1.1) or death from any cause
Measured: up to 24 months
Overall survival (OS)
Overall survival (OS) defined as the time from the date of first treatment to the date of death from any cause
Measured: up to 24 months
Incidence of treatment-related adverse events (TRAEs)
Defined as the proportion of patients experiencing ≥1 adverse event deemed at least possibly related to the investigational therapy
Measured: up to 24 months
Incidence of Grade ≥3 TRAEs
Measured: up to 24 months
Change in serum bilirubin levels (direct) over time
Measured: Baseline, upon eligibility for Chemoimmunotherapy (up to 24 months)
Time to biliary decompression or normalization of bilirubin (total)
Measured: Baseline, upon eligibility for Chemoimmunotherapy (up to 24 months)
Time to biliary decompression or normalization of bilirubin (direct)
Measured: Baseline, upon eligibility for Chemoimmunotherapy (up to 24 months)
Percent of patients rendered eligible for chemoimmunotherapy with GCD/P
defined as total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN)
Measured: up to 24 months
Time to start of chemoimmunotherapy with GCD/P
Measured: up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2026
- Study start5 Oct 2026
- Primary completionSep 2027 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Biliary Track CancerStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kristen Spencer, DO, MPH, Principal investigator, NYU Langone Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07853690
- Sponsor's protocol ID
- 25-00631
- Status last verified
- Sep 2026
- First submitted
- 23 Sep 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Yes: The de-identified participant data from the final research dataset will be shared upon reasonable request beginning 9 to 36 months after publication or as required by a condition of awards or supporting agreements, provided the requesting investigator executes a data use agreement with NYU Langone Health. This instance of data sharing will also require separate IRB review as well as review from NYU Langone's Data Sharing Strategy Board (DSSB). Requests should be directed to: Kristen.spencer@nyulangone.org. The protocol and statistical analysis plan will be posted on Clinicaltrials.gov only as required by federal regulation or supporting awards and agreements.