Histotripsy for Cholangiocarcinoma
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new method called histotripsy, combined with chemoimmunotherapy, for people with biliary tract cancer (BTC), which affects parts of the bile duct system. The goal is to determine if this approach is safe and effective, potentially leading to more treatment options. Participants should have BTC that cannot be surgically removed and have not received prior treatment for advanced disease. Those with bile flow issues due to their cancer might also qualify.
As an unphased trial, this study provides a unique opportunity to explore innovative treatments that could expand future options for BTC patients.
What prior data suggests that histotripsy is safe for biliary tract cancer?
Research has shown that histotripsy, a new treatment under study for bile duct cancer, appears safe. In one study, histotripsy reduced tumors by an average of 73% in just 26 days, with no serious side effects reported. Another study demonstrated a 95% success rate, with only about 6.8% experiencing major complications, indicating it is generally well-tolerated with few side effects.
The chemoimmunotherapy drugs used in the study—cisplatin, gemcitabine, durvalumab, and pembrolizumab—are already approved for treating other types of cancer. While they can cause side effects, these are usually known and managed by doctors.
Overall, evidence suggests that histotripsy and these drugs are reasonably safe. However, since everyone reacts differently to treatments, discussing expectations with a doctor is important.12345Why are researchers excited about this trial?
Histotripsy is unique because it offers a non-invasive way to target and break down tumors using focused ultrasound waves, potentially reducing the need for surgery and minimizing side effects. Unlike standard chemotherapy and immunotherapy, which can affect both healthy and cancerous cells throughout the body, histotripsy precisely targets the tumor itself, potentially leading to fewer systemic side effects. Researchers are excited about this approach as it could improve treatment outcomes for cholangiocarcinoma by enhancing the effectiveness of existing therapies like chemoimmunotherapy, offering a new avenue for patients who are not initially eligible for such treatments.
What evidence suggests that histotripsy could be an effective treatment for biliary tract cancer?
Research has shown that combining chemotherapy drugs gemcitabine and cisplatin with immunotherapy drugs like durvalumab or pembrolizumab may effectively treat biliary tract cancer. In this trial, participants in Cohort 1 will receive this combination therapy alongside histotripsy. Specifically, studies have demonstrated that patients lived between 4.6 to 11.7 months on average, with some experiencing tumor reduction. One study found that 72% of patients saw their cancer shrink. The side effects were generally manageable, making this treatment a promising option. These findings suggest that this combination of chemotherapy and immunotherapy could effectively treat biliary tract cancer.678910
Who Is on the Research Team?
Kristen Spencer, DO, MPH
Principal Investigator
NYU Langone Health
Are You a Good Fit for This Trial?
This trial is for adults with biliary tract cancer (cholangiocarcinoma). Participants should be able to safely undergo the study procedures and receive chemoimmunotherapy. Specific inclusion or exclusion details are not provided, but generally people must be healthy enough for treatment.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Histotripsy is added to chemoimmunotherapy regime with gemcitabine, cisplatin, and durvalumab/pembrolizumab in patients with untreated locally advanced/metastatic BTCs. Peripheral blood sampling and imaging assessment will be done at baseline and after every 4 treatment cycles.
Follow-up
Participants are monitored for safety and effectiveness after treatment, including monitoring for serious adverse events and disease progression.
Extension
Patients 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.
What Are the Treatments Tested in This Trial?
Interventions
- Cisplatin
- Durvalumab
- Gemcitabine
- Histotripsy
- Pembrolizumab
Trial Overview
The study is testing a new technique called histotripsy (using focused ultrasound waves to break up tumors) combined with standard chemotherapy (gemcitabine, cisplatin) and immunotherapy drugs (durvalumab or pembrolizumab). The main goal is to see if this combination is safe and doable.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
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.
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
NYU Langone Health
Lead Sponsor
Citations
Gemcitabine Plus Cisplatin for Advanced Biliary Tract Cancer
Median overall survival ranged from 4.6 to 11.7 months, and response rate ranged from 17.1% to 36.6%. Toxicities were generally acceptable and manageable.
The DEBATE Trial: Neoadjuvant Durvalumab Plus GemCis ...
Overall survival was 6.2 (95% CI, 2.7-NA) months in the durvalumab plus GemCis arm and 25.6 (95% CI, 15.1-NA) months in the GemCis arm.
Efficacy and Toxicity Profile of Carboplatin/Gemcitabine ... - PMC
Of the 66 patients studied, median overall survival was 8.97 months (95% CI: 6.78–11.16), and median progression-free survival was 5.88 months ( ...
Safety and Preliminary Activity of Ivosidenib Plus ...
Real‑world outcomes show survival benefit of first-line durvalumab or pembrolizumab plus gemcitabine and cisplatin in advanced biliary tract cancer. Read ...
Durvalumab + Chemotherapy for Bile Duct Cancer
Research has shown that this combination may be effective, with one study reporting a 72% objective response rate, indicating that many patients' tumors shrank ...
6.
fusfoundation.org
fusfoundation.org/posts/histotripsy-for-liver-tumors-one-year-data-suggest-90-tumor-control/Histotripsy for Liver Tumors: One-Year Data Suggest 90% ...
Overall survival at one year was 73.3% histotripsy can avoid damage to blood vessels and bile ducts within the treated area.
Histotripsy for the Treatment of Cholangiocarcinoma in a ...
Histotripsy achieved an average of 73% reduction of tumor diameter 26 d after treatment, with no significant adverse events. histotripsy can be used as a safe ...
Histotripsy for the Treatment of Cholangiocarcinoma Liver ...
Overall, the results of this study suggest that histotripsy has the potential to be used for the ablation of CC tumors while also highlighting the need for ...
9.
ccanewsonline.com
ccanewsonline.com/issues/2025/december-2025-vol-6-no-4/the-potential-of-histotripsy-in-the-treatment-of-cholangiocarcinomaThe Potential of Histotripsy in the Treatment ...
The study reported 95.5% technical success, 90.0% local tumor control at 1 year, and had low rates (6.8%) of major complications.1 Additionally, real-world data ...
Histotripsy and cholangiocarcinoma - AMMF
The overall survival was 83% and 58.6% at six- and twelve-months respectively. Safety: The most commonly reported adverse effects of the treatment were ...
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