Open pilot study of treatment with an antibiotic called vancomycin to children and adolescents with chronic inflammation of the bile ducts, called primary sclerosing cholangitis (PSC)

Conditions: Primary Sclerosing Cholangitis (PSC) is a progressive disease of the biliary system which might lead to increased risk of cholangiocancer. PSC constitutes a common indication for adult liver transplantation (ltx). In 80% of cases PSC is associated with inflammatory bowel disease (IBD), while 3-5 % of patients with colonic IBD, suffer from the disease. The conservative treatment options are few. There is a relatively high risk of recurrency after ltx.
Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Registration Number: EUCTR2009-018034-11-SE

Lead Sponsor: Karolinska Institutet

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 20

Inclusion Criteria

Children and adolescents > 1 year < 20 years who have MRC verified PSC and markers of disease which can easily be followed until normalisation:
- jaundice, itching and ascites
- increased liver function tests
- The initial liver biopsy consistent with PSC, not compulsory.
- Colonoscopy
The patients should be newly diagnosed or therapy resistant to commonly used therapy.

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Known hypersensitivity towards vancomycin
Pregnancy
Chronic renal failure.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To study if oral vancomycin during 1 year may improve liver- and gut function. ;Secondary Objective: To study if oral vancomycin during 1 year may improve liver histology and magnetic resonance cholangiography (MRC);Primary end point(s): Clinical status of patients after 1 year<br>Improvement of symptoms after 1 year<br>Improvement of liver function tests after 1 year<br>Improvement of bile duct stenosis as measured by MRC after 1 year<br>Improvement of liver histology after 1 year;Timepoint(s) of evaluation of this end point: One year after treatment start

Secondary Outcome Measures