NCT07477782

Fecal Microbiota Transplantation for Primary Sclerosing Cholangitis - Randomized Study Versus Sham Transplantation

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 72 (estimated)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

Primary Sclerosing Cholangitis (PSC) is a rare cholestatic liver disease, commonly associated with inflammatory bowel disease (IBD) The aim of the present trial is to assess the efficacy of fecal microbiota transplantation (FMT) on ALP and bilirubin compared to sham transplantation in addition to ursodeoxycholic acid (UDCA) treatment in PSC patients.

Detailed description

Primary Sclerosing Cholangitis (PSC) is a rare cholestatic liver disease, commonly associated with inflammatory bowel disease (IBD) and characterized by progressive obliterative fibrosis of the biliary tree. PSC can lead to cirrhosis, end-stage liver disease, and hepatobiliary and colorectal cancer. Serum levels of alkaline phosphatase (ALP) and bilirubin are markers of cholestasis and have a prognostic value in PSC. Liver transplantation is the only validated treatment since no medication has been reported to improve survival of PSC patients. However, ursodeoxycholic acid (UDCA), which has shown efficacy to improve liver tests, notably ALP, is approved for treatment of PSC and is prescribed in all PSC patients in France. Several studies have demonstrated that the gut microbiota plays an important role in the pathogenesis and the progression of PSC. First, PSC patients display an intestinal dysbiosis, regardless of IBD status. This dysbiosis is characterized by a decrease in diversity and some changes in the composition of gut microbiota. Second, fecal microbiota transplantation (FMT) from PSC patients into mice aggravate the cholestatic liver disease, suggesting an impact of the gut microbiota on PSC. Third, modulation of gut microbiota by antibiotic therapy can improve liver tests in PSC patients. A recent uncontrolled study, performed in 10 PSC patients, showed that FMT was safe. Moreover, in this study, a single FMT was associated with a reduction of ALP levels in 33% patients. FMT also increased bacterial diversity in all patients, and abundance of engrafted bacteria in patients post-FMT tended to be correlated with decreased ALP levels. Thus, FMT could be a useful therapy in PSC patients. The aim of the present trial is to assess the efficacy of FMT on ALP and bilirubin compared to sham transplantation in addition to UDCA treatment in PSC patients.

Conditions

• Primary Sclerosing Cholangitis (PSC)
• Inflammatory Bowel Disease (IBD)

Interventions

Type	Name	Description
DRUG	Fecal microbiota transplantation (FMT)	One to 4 weeks after randomization, the patient will be hospitalized in one of the hepato-gastroenterology department involved in the study for the colonoscopy. The patient will then receive either FMT (suspension of 50g of stools in 300ml of cryopreservative solution) in the terminal ileum or the caecum. At W12 and W24 after first colonoscopy , the patient will receive orally 20 FMT (capsules swallowed in front of a physician or a nurse in hospital)
DRUG	Sham-transplantation (placebo)	One to 4 weeks after randomization, the patient will be hospitalized in one of the hepato-gastroenterology department involved in the study for the colonoscopy. The patient will then receive sham transplantation (FMT vehicle, i.e. 300ml of cryopreservative solution) in the terminal ileum or the caecum. At W12 and W24 after first colonoscopy , the patient will receive orally 20 sham capsules (capsules swallowed in front of a physician or a nurse in hospital)

Timeline

• Start date: 2026-05-01
• Primary completion: 2029-04-01
• Completion: 2030-05-01
• First posted: 2026-03-17
• Last updated: 2026-03-17

Locations

1 site across 1 country: France