Vancomycin for Primary Sclerosing Cholangitis

NCT03710122 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: 18-003439FD-R-6102
• Study Type: interventional
• Target: 102
• Locations: 1 country
• Sites: 4

Brief Summary

To find out if vancomycin is a safe and effective therapy for primary sclerosing cholangitis. Funding Source - FDA OOPD

Conditions

Primary Sclerosing Cholangitis

Outcome Measures

Primary Outcomes (5)

• Alkaline Phosphatase at 6 Months

  Determine if OV normalizes serum ALP in adults with PSC. Levels of serum ALP obtained at 6 months of OV treatment, and will be compared to those obtained at baseline (month 0), and with values at the same study time points in the placebo arm.

  6 months

• Alkaline Phosphatase at 12 Months

  Determine if OV normalizes serum ALP in adults with PSC. Levels of serum ALP obtained at 12 months of OV treatment, and will be compared to those obtained at baseline (month 0), and with values at the same study time points in the placebo arm.

  12 months

• Alkaline Phosphatase at 18 Months

  Determine if OV normalizes serum ALP in adults with PSC. Levels of serum ALP obtained at 18 months of OV treatment, and will be compared to those obtained at baseline (month 0), and with values at the same study time points in the placebo arm.

  18 months

• Alkaline Phosphatase at 21 Months

  To determine if there is a change in alkaline phosphatase 3 months after study treatment completed

  21 months

• Alkaline Phosphatase at 24 Months

  To determine if there is a change in alkaline phosphatase 6 months after stopping study treatment

  24 months

Secondary Outcomes (1)

• Number of Participants With Reduction in Liver Stiffness

  18 months

Study Arms (2)

Vancomycin

EXPERIMENTAL

Drug: Vancomycin

Placebo

PLACEBO COMPARATOR

Other: Placebo

Interventions

Vancomycin DRUG

Firvanq by Azurity Pharmaceuticals, Inc.

Vancomycin

Placebo OTHER

Placebo for Vancomycin

Placebo

Eligibility Criteria

• Age: 18 Years - 76 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female subject age 18-76 years
• Diagnosis of PSC consistent with the guidelines published by the American Association for the Study of Liver Diseases (AASLD).39 All subjects must have an elevated serum ALP of at least 1.5 times upper limit of normal at baseline plus cholangiographic evidence of PSC, as demonstrated by magnetic resonance imaging, endoscopic retrograde cholangiography, direct cholangiography, or liver biopsy.
• Total bilirubin at screening must be ≤ 2 times upper limit of normal
• An ultrasound (or equivalent imaging modality) that excludes biliary obstruction and malignancy within 6 months of study entry,
• If a patient is on any of the following medications and/or supplements, he or she is expected to remain on the same daily dose through the treatment period: UDCA, azathioprine, prednisone (or an equivalent steroid compound), methotrexate, a 5-aminosalicylic acid, biologic therapy, and/or a probiotic.
• If a patient has been on obeticholic acid or other experimental therapies for PSC, they must complete a 3 month washout period before study entry
• PSC with or without inflammatory bowel disease, such as ulcerative colitis or Crohn's disease
• Must agree to comply with the study protocol and provide informed consent.

You may not qualify if:

• Administration of an antibiotic within 3 months prior to the study,
• Pregnancy or attempting to become pregnant or breastfeeding,
• Presence of any of the following:
• i. Hepatitis B infection
• ii. Hepatitis C infection (antibody positive); patients with a history of hepatitis C infection will be eligible for this study if they have undetectable levels of HCV RNA
• iii. Other cholestatic liver diseases such as primary biliary cholangitis and cholestatic diseases of pregnancy
• iv. Metabolic liver diseases such as Wilson's disease and hemochromatosis
• v. Inherited diseases of the liver such as α-1 antitrypsin deficiency
• vi. Immunoglobulin G4-related cholangitis
• vii. PSC with concomitant autoimmune hepatitis (AIH) and/or primary biliary cholangitis (previously known as primary biliary cirrhosis)
• viii. Secondary sclerosing cholangitis (SSC),
• ix. Active acute ascending cholangitis requiring antibiotics
• x. CCA (malignant biliary stricture, neoplasm, and cytology/histopathology or positive fluorescence in situ hybridization (FISH) consistent with adenocarcinoma of the bile duct)
• xi. A liver biopsy, if one has been previously obtained, which showed non-alcoholic steatohepatitis (NASH). Patients with suspected fatty liver by imaging will not be excluded
• xii. Presence of decompensated cirrhosis such as hepatic encephalopathy, hepato-renal syndrome and hepato-pulmonary syndrome,

Sponsors & Collaborators

• Elizabeth Carey: lead
• Arizona State University: collaborator

Study Sites (4)

Mayo Clinic Arizona

Phoenix, Arizona, 85259, United States

Location

Arizona State University

Tempe, Arizona, 85281, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Cholangitis, Sclerosing

Interventions

Vancomycin

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins

Limitations and Caveats

This study has several limitations. First, the trial was limited by patient withdrawal and early termination due to slowed recruitment. Second, given the sample size the subgroup analyses should be considered hypothesis generating. Third, there is a lack of validated surrogate endpoints for PSC.

Results Point of Contact

• Title: Elizabeth Carey
• Organization: Mayo Clinic Arizona

carey.elizabeth@mayo.edu

4803422000

Study Officials

• Elizabeth Carey, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: October 15, 2018
• First Posted: October 17, 2018
• Study Start: January 23, 2020
• Primary Completion: March 3, 2025
• Study Completion: March 20, 2025
• Last Updated: April 22, 2026
• Results First Posted: April 22, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)