Cholangiocarcinoma treatment with radiofrequency ablation or photodynamic therapy

The primary objective of this study is to determine whether **photodynamic therapy (PDT)** or **radiofrequency ablation (RFA)** leads to longer survival in patients with **hilar cholangiocarcinoma**. This is clinically relevant as it aims to identify the more effective treatment modality in extending patient survival, which is a critical outcome in the management of this aggressive cancer.

Secondary objectives include comparing the quality of life and quality-adjusted life years between PDT and RFA. Additionally, the study seeks to identify the most relevant harms associated with each treatment and their frequency of occurrence. These secondary objectives are important for understanding the broader impact of each treatment on patient well-being and safety.

The clinical trial focuses on participants diagnosed with **hilar cholangiocarcinoma**, confirmed either cytologically or histologically. The study population includes both male and female subjects aged 18 years and older. Participants are required to provide written informed consent, and surgery is not planned for these individuals. The trial does not involve a vulnerable population. The sponsor has not provided information regarding the total number of participants. Lifestyle considerations such as diet, physical activity, or habits are not specified in the available data. The selection criteria ensure that the study population is representative of adults with the specified medical condition, without planned surgical intervention.

The clinical trial is designed to evaluate the efficacy of **photodynamic therapy** (PDT) and radiofrequency ablation (RFA) in patients with hilar cholangiocarcinoma. This is a randomized, controlled, and double-blind study aimed at determining whether PDT or RFA leads to longer survival. The trial is categorized as a Phase IV study and is considered low intervention. The primary endpoint is overall survival, measured from randomization to death from any cause or the last known date the patient was alive, with comparisons between treatment arms based on the hazard ratio estimated from a Cox regression model. Secondary endpoints include overall survival from complementary perspectives, days alive and out of the hospital up to two years, quality of life at various time points, quality-adjusted life years, stent patency, laboratory parameters, and pruritus over two years.

The trial is expected to run from June 1, 2022, to December 31, 2027. Participants will be involved in the study for a maximum treatment period of one year. The study includes an initial screening visit to confirm eligibility, which requires a cytological or histological confirmation of hilar cholangiocarcinoma, no planned surgery, age 18 or older, and written informed consent. Follow-up visits will occur at specified intervals to assess quality of life and other secondary endpoints, with the end-of-study visit marking the conclusion of the participant's involvement. Conditions that may lead to early termination from the study include withdrawal of consent or any adverse events that compromise participant safety.

The clinical trial involves the use of **Porfimer Sodium**, a photosensitizer, as an experimental medication. **Porfimer Sodium** is administered in the form of an infusion. The pharmaceutical form is designated as PHF00231MIG. The dosage is calculated based on body weight, with a maximum daily dose of 2 mg/kg. The administration route is via infusion, and the treatment period is limited to a maximum of one day. The active substance, **Porfimer Sodium**, is a polymer-based compound, and its use in this trial is not part of a pediatric formulation. Participant compliance with the dosing schedule will be monitored throughout the study.

In addition to **Porfimer Sodium**, the trial also includes the use of **Foscan 1 mg/ml solution for injection**. This solution is administered via injection, with a maximum daily dose of 0.15 mg/kg. The pharmaceutical form is a solution for injection, and the treatment period is similarly restricted to one day. **Foscan** is provided by BIOLITEC PHARMA LTD. and is not a pediatric formulation. The administration of this solution will be closely monitored to ensure adherence to the dosing regimen.

Both **Porfimer Sodium** and **Foscan** are utilized as part of the study's objective to evaluate the efficacy of photodynamic therapy in the treatment of cholangiocarcinoma. The trial aims to determine whether photodynamic therapy or radiofrequency ablation leads to longer survival in patients. The study does not include any additional non-experimental treatments such as standard-of-care therapy or placebo. Compliance with the administration protocols and dosing schedules will be rigorously monitored to ensure the integrity of the trial data.

Efficacy in the clinical trial for the treatment of **cholangiocarcinoma** will be assessed using both primary and secondary endpoints. The primary endpoint is overall survival, defined as the time from randomization to death from any cause or to the last known date on which the patient was alive. This will be analyzed using a hazard ratio estimated from a Cox regression model to compare the treatment arms.

Secondary endpoints include several measures to provide complementary perspectives on efficacy. These include overall survival from different perspectives, days alive and out of hospital up to two years, and Quality of Life (QoL) assessments at various time points after randomization, specifically at 14 days (V2), approximately 3 months (V3), and as a function of time after 6 months, with adjustments for baseline values. Quality-adjusted life years (QALYs) will also be evaluated. Additional secondary endpoints involve stent patency assessed by clinicians through indicators such as cholangitis, cholestasis, and ultrasound, as well as laboratory parameters and pruritus as a function of time up to two years.