Clinical trial NCT01695174: A Pilot Study of Xifaxan to Treat Patients With PSC
Official title: A Pilot Study of Xifaxan in Patients With Primary Sclerosing Cholangitis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Primary Sclerosing Cholangitis (PSC)
Keywords: Primary Sclerosing Cholangitis
Xifaxan
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2012-09-27
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Improvement in alkaline phosphatase
timeFrame: Three months
measure: Absence of treatment failure
timeFrame: Three months
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic in Rochester
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Jayant Talwalkar, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Tabibian JH, Gossard A, El-Youssef M, Eaton JE, Petz J, Jorgensen R, Enders FB, Tabibian A, Lindor KD. Prospective Clinical Trial of Rifaximin Therapy for Patients With Primary Sclerosing Cholangitis. Am J Ther. 2017 Jan/Feb;24(1):e56-e63. doi: 10.1097/MJT.0000000000000102.