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A Pilot Study of Xifaxan to Treat Patients With PSC (PSC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01695174
Recruitment Status : Completed
First Posted : September 27, 2012
Last Update Posted : October 15, 2014
Information provided by (Responsible Party):
Jayant A. Talwalkar, Mayo Clinic

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.

Condition or disease Intervention/treatment Phase
Primary Sclerosing Cholangitis (PSC) Drug: Xifaxan Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study of Xifaxan in Patients With Primary Sclerosing Cholangitis
Study Start Date : August 2012
Actual Primary Completion Date : December 2013
Actual Study Completion Date : March 2014

Arm Intervention/treatment
Experimental: Xifaxan
Xifaxan 550 mg two times per day for three months
Drug: Xifaxan

Primary Outcome Measures :
  1. Improvement in alkaline phosphatase [ Time Frame: Three months ]
    An improvement in elevated levels of alkaline phosphatase to less than half of the initial level at study entry

  2. Absence of treatment failure [ Time Frame: 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01695174

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United States, Minnesota
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
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Principal Investigator: Jayant Talwalkar, MD Mayo Clinic
Publications of Results:
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Responsible Party: Jayant A. Talwalkar, Principal Investigator, Mayo Clinic Identifier: NCT01695174    
Other Study ID Numbers: 11-006516
First Posted: September 27, 2012    Key Record Dates
Last Update Posted: October 15, 2014
Last Verified: October 2014
Keywords provided by Jayant A. Talwalkar, Mayo Clinic:
Primary Sclerosing Cholangitis
Additional relevant MeSH terms:
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Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Gastrointestinal Agents