Primary Sclerosing CholangitisPhase 2/3NCT03710122

Vancomycin for Primary Sclerosing Cholangitis

A Phase 2/3 study testing Vancomycin in people with Primary Sclerosing Cholangitis, run by Elizabeth Carey at 4 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

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

Who is it for?: Ages 18 to 76 years
Conditions the study is for: Primary Sclerosing Cholangitis
What is being tested?: Vancomycin
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 102 participants
Where: 4 sitesUnited States
Study dates: 23 Jan 2020to 20 Mar 2025
Phase: Phase 2/3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 76 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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:
Hepatitis B infection
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
Other cholestatic liver diseases such as primary biliary cholangitis and cholestatic diseases of pregnancy

Show all 21Show fewer

Metabolic liver diseases such as Wilson's disease and hemochromatosis
Inherited diseases of the liver such as α-1 antitrypsin deficiency
Immunoglobulin G4-related cholangitis
PSC with concomitant autoimmune hepatitis (AIH) and/or primary biliary cholangitis (previously known as primary biliary cirrhosis)
Secondary sclerosing cholangitis (SSC),
Active acute ascending cholangitis requiring antibiotics
CCA (malignant biliary stricture, neoplasm, and cytology/histopathology or positive fluorescence in situ hybridization (FISH) consistent with adenocarcinoma of the bile duct)
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
Presence of decompensated cirrhosis such as hepatic encephalopathy, hepato-renal syndrome and hepato-pulmonary syndrome,
History of liver transplantation, anticipated need for liver transplantation within 12 months from randomization, or a Model of End Stage Liver Disease (MELD) score of ≥15
Ongoing alcohol abuse (>4 drinks per day for men, and >2 drinks per day for women)
History of allergic reaction to vancomycin,
Moderate-to-severe renal impairment with a calculated creatinine clearance of < 60mL/min
HIV/AIDS,
xviii. Any other conditions or abnormalities that, in the opinion of the investigator, may compromise the safety of the subject or interfere with the subject participating in or completing the study.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

Mayo Clinic Florida
Jacksonville, Florida, 32224
Mayo Clinic Arizona
Phoenix, Arizona, 85259
Mayo Clinic Rochester
Rochester, Minnesota, 55905
Arizona State University
Tempe, Arizona, 85281

Other trials in United States

About this study

Official title: A Prospective, Randomized, Multi-centered, Placebo-controlled Clinical Trial of Oral Vanycomycin in Adults With Primary Sclerosing Cholangitis

To find out if vancomycin is a safe and effective therapy for primary sclerosing cholangitis.

Funding Source - FDA OOPD

Read the detailed description

Determine if OV normalizes serum ALP in adults with PSC. Levels of serum ALP obtained at 6,12,and 18 months of OV treatment, and at 3, and 6 months post OV treatment will be compared to those obtained at baseline (month 0), and with values at the same study time points in the placebo arm.
Determine if OV stabilizes or improves liver fibrosis assessed by LSM using TE. Liver stiffness will be measured at 6, 12, and 18 months of OV treatment, and at 6 months post OV treatment, and values will be compared to those obtained at baseline (month 0), and with values in the placebo arm.
Determine the changes in the intestinal microbiota in relation to the use of OV, and study the correlation between the changes in the intestinal microbiota and the changes in: 1) liver enzymes, particularly serum ALP, and 2) liver stiffness, assessed by LSM using TE.
Determine if changes in proinflammatory cytokines (TGF-β, IL-4, IL-13, IL-10, etc.) predict response to OV. Cytokines will be measured at baseline, months 6, 12, 18, and at 3, and 6 months post OV treatment, if the study is positive.

Conditions: Primary Sclerosing Cholangitis
Being tested: Vancomycin, Placebo
Medical subject headings: Cholangitis, Sclerosing

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

VancomycinDrug
Firvanq by Azurity Pharmaceuticals, Inc.
PlaceboOther
Placebo for Vancomycin

Study groups in NCT03710122
Study group	Role	What they receive
Vancomycin	Experimental	Not described Receives: Drug: Vancomycin
Placebo	Placebo comparator	Not described Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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.
Measured: 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.
Measured: 18 months
Alkaline Phosphatase at 21 Months
To determine if there is a change in alkaline phosphatase 3 months after study treatment completed
Measured: 21 months
Alkaline Phosphatase at 24 Months
To determine if there is a change in alkaline phosphatase 6 months after stopping study treatment
Measured: 24 months

Secondary outcomes (1)

Number of Participants With Reduction in Liver Stiffness
Statistically significant reduction in liver stiffness measurement (kPa)
Measured: 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

23 Jan 202020 Mar 2025

First posted17 Oct 2018
Study start23 Jan 2020
Primary completion3 Mar 2025
Study completion20 Mar 2025

Why it stopped: Funding terminated

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 102 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03710122
Sponsor's protocol ID: 18-003439
Other IDs: FD-R-6102
Status last verified: Sep 2026
First submitted: 15 Oct 2018
Last updated: 21 Oct 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

Eaton JE, Clayton MW, Harnois DM, Butterfield DJ 3rd, Zhang N, Venkatesh SK, Nguyen NV, Gossard AA, Lindor KD, LaRusso NF, Carey EJ. Oral Vancomycin Is Not Associated With Meaningful Changes in Liver-Related End Points Among Adults With Primary Sclerosing Cholangitis: A Randomized, Placebo-Controlled Trial. Am J Gastroenterol. 2026 Jul 23. doi: 10.14309/ajg.0000000000004135. Online ahead of print.
PubMed 42489629

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Active, not recruitingPhase 2
A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.
Primary Sclerosing Cholangitis
Active, not recruitingPhase 2
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
Primary Sclerosing Cholangitis
RecruitingObservational study
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach
Primary Sclerosing Cholangitis
Not yet recruitingObservational study
PET/MRI of Primary Sclerosing Cholangitis
PSC

All Primary Sclerosing Cholangitis trials

Study at a glance

From the registry record, last updated by the sponsor on 21 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Prospective, Randomized, Multi-centered, Placebo-controlled Clinical Trial of Oral Vanycomycin in Adults With Primary Sclerosing Cholangitis
ClinicalTrials.gov ID: NCT03710122
Acronym / sponsor ID: 18-003439
Conditions: Primary Sclerosing Cholangitis
Phase: Phase 2/3
Study type: Interventional
Recruitment status: TerminatedReason stopped: Funding terminated
Enrolment: 102 participants (actual)
Lead sponsor: Elizabeth CareyUniversity, hospital or other
Collaborators: Arizona State University
Study start: 23 Jan 2020 (actual)
Primary completion: 3 Mar 2025 (actual)
Study completion: 20 Mar 2025 (actual)
First posted: 17 Oct 2018 (actual)
Results: Posted 22 Apr 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

To find out if vancomycin is a safe and effective therapy for primary sclerosing cholangitis.

Funding Source - FDA OOPD

Detailed description

Determine if OV normalizes serum ALP in adults with PSC. Levels of serum ALP obtained at 6,12,and 18 months of OV treatment, and at 3, and 6 months post OV treatment will be compared to those obtained at baseline (month 0), and with values at the same study time points in the placebo arm.
Determine if OV stabilizes or improves liver fibrosis assessed by LSM using TE. Liver stiffness will be measured at 6, 12, and 18 months of OV treatment, and at 6 months post OV treatment, and values will be compared to those obtained at baseline (month 0), and with values in the placebo arm.
Determine the changes in the intestinal microbiota in relation to the use of OV, and study the correlation between the changes in the intestinal microbiota and the changes in: 1) liver enzymes, particularly serum ALP, and 2) liver stiffness, assessed by LSM using TE.
Determine if changes in proinflammatory cytokines (TGF-β, IL-4, IL-13, IL-10, etc.) predict response to OV. Cytokines will be measured at baseline, months 6, 12, 18, and at 3, and 6 months post OV treatment, if the study is positive.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 2

Arm	Type	Receives
Vancomycin	Experimental	Drug: Vancomycin
Placebo	Placebo comparator	Other: Placebo

Who may qualify

Ages: 18 to 76 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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.

Key exclusion criteria (21 listed)

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:
xviii. Any other conditions or abnormalities that, in the opinion of the investigator, may compromise the safety of the subject or interfere with the subject participating in or completing the study.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
VancomycinFirvanq by Azurity Pharmaceuticals, Inc.	Drug	Vancomycin
PlaceboPlacebo for Vancomycin	Other	Placebo

Primary outcome

Alkaline Phosphatase at 6 Months
Time frame: 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.
Alkaline Phosphatase at 12 Months
Time frame: 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.
Alkaline Phosphatase at 18 Months
Time frame: 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.
Alkaline Phosphatase at 21 Months
Time frame: 21 months
To determine if there is a change in alkaline phosphatase 3 months after study treatment completed
Alkaline Phosphatase at 24 Months
Time frame: 24 months
To determine if there is a change in alkaline phosphatase 6 months after stopping study treatment

1 secondary outcome

Number of Participants With Reduction in Liver Stiffness
Time frame: 18 months

Results and safety

Results posted to the registry on 22 Apr 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Vancomycin	40	15
Placebo	42	20

Primary outcome: Alkaline Phosphatase at 6 Months

Time frame: 6 months. Mean (Inter-Quartile Range). Unit: U/L

Measure	Vancomycin n = 24	Placebo n = 36
Value	351 (275 to 407)	355 (300 to 410)

Primary outcome: Alkaline Phosphatase at 12 Months

Time frame: 12 months. Mean (Inter-Quartile Range). Unit: U/L

Measure	Vancomycin n = 19	Placebo n = 25
Value	280 (210 to 356)	354 (280 to 415)

Primary outcome: Alkaline Phosphatase at 18 Months

Time frame: 18 months. Mean (Inter-Quartile Range). Unit: U/L

Measure	Vancomycin n = 15	Placebo n = 20
Value	304 (255 to 311)	311 (304 to 446)

Primary outcome: Alkaline Phosphatase at 21 Months

Time frame: 21 months. Mean (Standard Deviation). Unit: U/L

Measure	Vancomycin n = 12	Placebo n = 16
Value	349.5 (160.9)	320.5 (159.7)

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Adverse events were monitored by patient contact every 3 months with the study coordinator and physician visits every 6 months from the time of enrolment until the end of follow-up (24 months). Additionally, patients were instructed to contact the study coordinator immediately when adverse events occurred.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Vancomycin	5 of 40 (12.5%)	8 of 40 (20%)	0 of 40
Placebo	7 of 42 (16.7%)	12 of 42 (28.6%)	0 of 42

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 Oct 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 42489629Eaton JE, Clayton MW, Harnois DM, Butterfield DJ 3rd, Zhang N, Venkatesh SK, Nguyen NV, Gossard AA, Lindor KD, LaRusso NF, Carey EJ. Oral Vancomycin Is Not Associated With Meaningful Changes in Liver-Related End Points Among Adults With Primary Sclerosing Cholangitis: A Randomized, Placebo-Controlled Trial. Am J Gastroenterol. 2026 Jul 23. doi: 10.14309/ajg.0000000000004135. Online ahead of print.
Mayo Clinic Clinical TrialsLink provided by the sponsor.
Individual participant dataSharing planned: No

Related trials for Primary Sclerosing Cholangitis

RecruitingPhase 3
A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Active, not recruitingPhase 2
A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.
Primary Sclerosing Cholangitis
Active, not recruitingPhase 2
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)
Primary Sclerosing Cholangitis
RecruitingObservational study
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach
Primary Sclerosing Cholangitis
Not yet recruitingObservational study
PET/MRI of Primary Sclerosing Cholangitis
PSC

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Elizabeth Carey, MD principal investigator

Mayo Clinic

4 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Vancomycin in people with Primary Sclerosing Cholangitis, run by Elizabeth Carey at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 21 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Alkaline Phosphatase at 6 Months (time frame: 6 months). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03710122, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Oct 2026.