Primary Sclerosing CholangitisPhase 1PSCNCT01085760
A Pilot Study of Vancomycin or Metronidazole in Patients With Primary Sclerosing Cholangitis
A Phase 1 study testing Vancomycin and Metronidazole in people with Primary Sclerosing Cholangitis, run by Mayo Clinic at 1 site in United States.
Trial summary
The purpose of this study is to determine whether Vancomycin or Metronidazole is safe and beneficial in the treatment of Primary Sclerosing Cholangitis. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- Vancomycin and Metronidazole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2010to Nov 2011
- Phase
- Phase 1
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of PSC established by alkaline phosphatase >1.5 times and/or two fold elevation of liver transaminases (AST and/or ALT) for at least 6 months duration.
- Cholangiography demonstrating intrahepatic and/or extrahepatic biliary obstruction, beading, or narrowing consistent with PSC.
- Both genders.
- Age ≥ 18 years old and < than 75 years old.
- Patient's informed consent for study participation.
Who cannot join
- Treatment with systematic antibiotics, azulfidine, ursodeoxycholic acid, corticosteroids, colchicine, methotrexate, azathioprine, cyclosporine, chlorambucil, budesonide, pentoxifylline, tacrolimus, silymarin or prednisone in the preceding three months.
- Active drug or alcohol use.
- Prior history of allergic reactions to the antibiotics which will be used in the study.
- Any condition that, in the opinion of the investigator, would interfere with the patients' 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.
- Pregnancy or current lactation. Subjects becoming pregnant during the study will be withdrawn.
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to determine whether Vancomycin or Metronidazole is safe and beneficial in the treatment of Primary Sclerosing Cholangitis.
Read the detailed description
In this protocol, we propose the assessment of potential beneficial effects of the antibiotics vancomycin and metronidazole on liver biochemistries, liver related symptoms and Mayo risk score in patients with PSC. The patients will be randomized into four groups of ten patients: one group will receive low dose vancomycin, one group will receive high dose vancomycin, one group will receive low dose metronidazole and one group will receive high dose metronidazole. Each group will be treated for three months.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Vancomycin, Metronidazole
- 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
Comparison of different doses of drug
VancomycinDrug
Comparison of different doses of drug
MetronidazoleDrug
Comparison of different doses of drug
MetronidazoleDrug
Comparison of different doses of drug
VancomycinDrug
Comparison of different drug doses
| Study group | Role | What they receive |
|---|---|---|
| Vancomycin 125 mg orally 4 times a day | Experimental | The patients will be randomized into four groups of ten patients: one group will receive low dose vancomycin, one group will receive high dose vancomycin, one group will receive low dose metronidazole and one group will receive high dose metronidazole. Receives: Drug: Vancomycin |
| Vancomycin 250 mg orally 4 times a day | Experimental | Not described Receives: Drug: Vancomycin |
| Metronidazole 250 mg orally 3 times a day | Experimental | Not described Receives: Drug: Metronidazole |
| Metronidazole 500 mg orally 3 times a day | Experimental | Not described Receives: Drug: Metronidazole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Alkaline Phosphatase Following 12 Weeks of Treatment
Measured: baseline, 12 weeks
Secondary outcomes (3)
Change From Baseline in Total Bilirubin Following 12 Weeks Treatment
Measured: baseline, 12 weeks
Change From Baseline in Mayo PSC Risk Score Following 12 Weeks of Treatment
The Mayo PSC risk score was calculated for each patient at baseline and at 12 weeks, where Risk = 0.03 (age [years]) + 0.54 Ln (total bilirubin [mg/dL]) + 0.54 Ln (AST [IU/L]) + 1.24 (variceal bleeding) - 0.84 (albumin [g/dL]). There is no range, minimum, or maximum value but greater values indicate worse disease.
Measured: baseline, 12 weeks
Change From Baseline in C-Reactive Protein Following 12 Weeks of Treatment
Measured: baseline, 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Mar 2010 (estimated)
- Study startFeb 2010
- Primary completionNov 2011
- Study completionNov 2011
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.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- PSC Partners Seeking a Cure
- Responsible party
- Principal investigator, Jayant A. Talwalkar, Mayo Clinic
- Investigators
- Jayant A Talwalkar, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01085760
- Sponsor's protocol ID
- 08-008247
- Acronym
- PSC
- Status last verified
- Jul 2013
- First submitted
- 10 Mar 2010
- Last updated
- 19 Sep 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Tabibian JH, Weeding E, Jorgensen RA, Petz JL, Keach JC, Talwalkar JA, Lindor KD. Randomised clinical trial: vancomycin or metronidazole in patients with primary sclerosing cholangitis - a pilot study. Aliment Pharmacol Ther. 2013 Mar;37(6):604-12. doi: 10.1111/apt.12232. Epub 2013 Feb 5.
PubMed 23384404
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Vancomycin or Metronidazole in Patients With Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT01085760
- Acronym / sponsor ID
- PSC / 08-008247
- Conditions
- Primary Sclerosing Cholangitis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Collaborators
- PSC Partners Seeking a Cure
- Study start
- Feb 2010
- Primary completion
- Nov 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 12 Mar 2010 (estimated)
- Results
- Posted 19 Sep 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine whether Vancomycin or Metronidazole is safe and beneficial in the treatment of Primary Sclerosing Cholangitis.
Detailed description
In this protocol, we propose the assessment of potential beneficial effects of the antibiotics vancomycin and metronidazole on liver biochemistries, liver related symptoms and Mayo risk score in patients with PSC. The patients will be randomized into four groups of ten patients: one group will receive low dose vancomycin, one group will receive high dose vancomycin, one group will receive low dose metronidazole and one group will receive high dose metronidazole. Each group will be treated for three months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Vancomycin 125 mg orally 4 times a day | Experimental | Drug: VancomycinThe patients will be randomized into four groups of ten patients: one group will receive low dose vancomycin, one group will receive high dose vancomycin, one group will receive low dose metronidazole and one group will receive high dose metronidazole. |
| Vancomycin 250 mg orally 4 times a day | Experimental | Drug: Vancomycin |
| Metronidazole 250 mg orally 3 times a day | Experimental | Drug: Metronidazole |
| Metronidazole 500 mg orally 3 times a day | Experimental | Drug: Metronidazole |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of PSC established by alkaline phosphatase >1.5 times and/or two fold elevation of liver transaminases (AST and/or ALT) for at least 6 months duration.
- Cholangiography demonstrating intrahepatic and/or extrahepatic biliary obstruction, beading, or narrowing consistent with PSC.
- Both genders.
- Age ≥ 18 years old and < than 75 years old.
- Patient's informed consent for study participation.
Key exclusion criteria (8 listed)
- Treatment with systematic antibiotics, azulfidine, ursodeoxycholic acid, corticosteroids, colchicine, methotrexate, azathioprine, cyclosporine, chlorambucil, budesonide, pentoxifylline, tacrolimus, silymarin or prednisone in the preceding three months.
- Active drug or alcohol use.
- Prior history of allergic reactions to the antibiotics which will be used in the study.
- Any condition that, in the opinion of the investigator, would interfere with the patients' ability to complete the study safely or successfully.
- Evidence of decompensated liver disease such as recurrent variceal bleeding, refractory ascites or spontaneous hepatic encephalopathy.
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 |
|---|---|---|
| VancomycinComparison of different doses of drug | Drug | Vancomycin 125 mg orally 4 times a day |
| VancomycinComparison of different doses of drug | Drug | Vancomycin 250 mg orally 4 times a day |
| MetronidazoleComparison of different doses of drug | Drug | Metronidazole 250 mg orally 3 times a day |
| MetronidazoleComparison of different doses of drug | Drug | Metronidazole 500 mg orally 3 times a day |
| VancomycinComparison of different drug doses | Drug | Vancomycin 125 mg orally 4 times a day |
Primary outcome
Change From Baseline in Alkaline Phosphatase Following 12 Weeks of Treatment
Time frame: baseline, 12 weeks
3 secondary outcomes
Change From Baseline in Total Bilirubin Following 12 Weeks Treatment
Time frame: baseline, 12 weeks
Change From Baseline in Mayo PSC Risk Score Following 12 Weeks of Treatment
Time frame: baseline, 12 weeks
Change From Baseline in C-Reactive Protein Following 12 Weeks of Treatment
Time frame: baseline, 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 23384404Tabibian JH, Weeding E, Jorgensen RA, Petz JL, Keach JC, Talwalkar JA, Lindor KD. Randomised clinical trial: vancomycin or metronidazole in patients with primary sclerosing cholangitis - a pilot study. Aliment Pharmacol Ther. 2013 Mar;37(6):604-12. doi: 10.1111/apt.12232. Epub 2013 Feb 5.
Related trials for Primary Sclerosing Cholangitis
RecruitingObservational study
Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach
Primary Sclerosing Cholangitis
Not yet recruitingPhase 2
FEAST and HOPE: Redox PSC Pilot Study
Primary Sclerosing Cholangitis (PSC)
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
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.
Jayant A Talwalkar, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Vancomycin and Metronidazole in people with Primary Sclerosing Cholangitis, run by Mayo Clinic at 1 site 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 5 inclusion and 8 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: finished, as of the sponsor's last update on 19 Sep 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 19 Sep 2013). 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?
Change From Baseline in Alkaline Phosphatase Following 12 Weeks of Treatment (time frame: baseline, 12 weeks). 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 NCT01085760, 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 19 Sep 2013.