Primary Sclerosing Cholangitis (PSC)
Conditions
Keywords
Primary Sclerosing Cholangitis, Xifaxan
Brief summary
In the current protocol, we propose the assessment of potential beneficial effects of the antibiotic Xifaxan on liver biochemistries, liver related symptoms and Mayo risk score in 15 adult and 5 pediatric patients with PSC. Adult patients will receive Xifaxan, 550 mg twice daily over a 12-week period. Pediatric patients with PSC whose weight is greater than or equal to 40 kg will receive Xifaxan, 550 mg twice daily.
Interventions
DRUGXifaxan
Sponsors
Mayo Clinic
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of PSC established by all of the following criteria: * Alkaline phosphatase \>1.5 times upper limit of normal for at least 6 months duration * Gamma-glutamyl transferase (GGT) \>1.5 times upper limit of normal in pediatric patients * Cholangiography demonstrating intrahepatic and/or extrahepatic biliary obstruction, beading, or narrowing consistent with PSC * Liver histology in the past (if available for review) with features consistent with or diagnostic of PSC * Both genders * Adults: Ages 18-75 years. * Pediatric: Weight \> 40 kg * Patient's informed consent for study participation
Exclusion criteria
* Treatment with systematic antibiotics, Azulfidine, ursodeoxycholic acid, corticosteroids, colchicine, methotrexate, azathioprine, cyclosporine, chlorambucil, budesonide, pentoxifylline, tacrolimus, vitamin E or prednisone in the preceding three months * Active drug or alcohol use * Prior history of allergic reaction to the antibiotics which will be used in the study * Any condition that, in the opinion of the investigator, would interfere with the patient's ability to complete the study safely or successfully * Evidence of decompensated liver disease such as recurrent variceal bleeding, refractory ascites or spontaneous hepatic encephalopathy * Anticipated need for transplantation in one year (Mayo survival model \<80% one-year survival without transplant) * Findings highly suggestive of liver disease of other etiology such as chronic alcoholic liver disease, chronic hepatitis B or C infection, hemochromatosis, Wilson's disease, 1-antitrypsin deficiency, non-alcoholic steatohepatitis, primary biliary cirrhosis or secondary sclerosing cholangitis * Treatment with any study medications in the preceding three months * Pregnancy or current lactation; subjects becoming pregnant during the study despite all the precautions will be withdrawn and referred to their primary physicians
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Improvement in alkaline phosphatase | Three months | An improvement in elevated levels of alkaline phosphatase to less than half of the initial level at study entry |
| Absence of treatment failure | Three months | Absence of treatment failure which is defined as any of the following: death, need for liver transplantation, side effects requiring discontinuation of therapy, worsening of liver biochemistries, voluntary discontinuation for any reason, marked worsening of fatigue or itching. |
Countries
United States
Outcome results
None listed