A Pilot Study of Xifaxan to Treat Patients With PSC

Mayo Clinic

Status and phase

Completed

Phase 1

Conditions

Primary Sclerosing Cholangitis (PSC)

Treatments

Drug: Xifaxan

Study type

Interventional

Funder types

Other

Identifiers

NCT01695174

11-006516

Details and patient eligibility

About

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.

Enrollment

16 patients

Sex

All

Ages

18 to 75 years old

Volunteers

No Healthy Volunteers

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