Organoids for Bile Leaks

NCT07214649 | Not Yet Recruiting Phase 2 FDA Drug

• 2 other identifiers: 2025001FPG
• Study Type: interventional
• Target: 25
• Locations: 0 countries
• Sites: N/A

Brief Summary

Background: Bile leakage remains a major complication after hepatobiliary surgery and liver transplantation. While most cases are managed through standard radiologic or endoscopic interventions, a subset of patients fails to respond and may face recurrent interventions or even retransplantation. Recent advances in regenerative medicine, particularly the development of extrahepatic cholangiocyte organoids (ECOs), offer a promising alternative. Objective: This prospective, 5-year study aims to evaluate the feasibility, safety, and efficacy of autologous ECO-based cell therapy to reconstruct bile ducts in patients with persistent bile leakage unresponsive to standard care. Methods: Patients undergoing hepatobiliary or liver transplant surgery will have a biopsy of extrahepatic bile duct tissue collected perioperatively. The tissue will be processed and stored in a dedicated biobank. Upon development of a refractory bile leak, ECOs will be generated from the stored tissue and delivered to the site of injury through radiological or endoscopic routes, as decided by a multidisciplinary team. Success will be evaluated by resolution of leakage (radiologically or endoscopically) and absence of further intervention. Significance: The use of patient-specific ECOs holds transformative potential. Organoids can be derived and expanded in vitro while maintaining cholangiocyte identity and function. Preclinical studies in murine and human models show successful engraftment and functional integration into biliary epithelium. Expected Outcomes: This project aims to pioneer a novel, minimally invasive, personalized regenerative therapy for otherwise intractable biliary complications.

Conditions

Biliary Anastomosis Complication

Keywords

biliary leaks; cholangiocyte organoids

Outcome Measures

Primary Outcomes (1)

• Treatment of the leaks

  Absence of leak at radiological or endoscopic evaluation

  1 week

Study Arms (1)

Organoid Arm

EXPERIMENTAL

Biological: Organoid-guided treatment

Interventions

Organoid-guided treatment BIOLOGICAL

Engrafment of the Cholangiocyte Organoids in the common bile duct with leakage

Organoid Arm

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age over 18 years old
• Undergone HPB surgical procedure with hepatic duct reconstruction
• Presence of anastomotic bile leakage untreatable with radiological or endoscopic approach
• Presence of bile duct specimen

You may not qualify if:

• Age under 18 years old
• Undergone HPB surgical procedure without hepatic duct reconstruction
• Absence of anastomotic bile leakage untreatable with radiological or endoscopic approach
• Absence of bile duct specimen

Sponsors & Collaborators

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS: lead

Central Study Contacts

Marco M Pascale, MD

CONTACT

0039 3888264297; pascale.marco.maria@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: October 3, 2025
• First Posted: October 9, 2025
• Study Start: January 1, 2026
• Primary Completion (Estimated): June 1, 2030
• Study Completion (Estimated): December 1, 2030
• Last Updated: October 9, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share