Primary Sclerosing Cholangitis (PSC)Phase 1PSCNCT01695174
A Pilot Study of Xifaxan to Treat Patients With PSC
A Phase 1 study testing Xifaxan in people with Primary Sclerosing Cholangitis (PSC), run by Mayo Clinic at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis (PSC)
- What is being tested?
- Xifaxan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2012to Mar 2014
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 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
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- Adults: Ages 18-75 years.
- Pediatric: Weight > 40 kg
- 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, 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)
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- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: A Pilot Study of Xifaxan in Patients With Primary Sclerosing Cholangitis
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.
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Being tested
- Xifaxan
- Keywords
- Primary Sclerosing Cholangitis, Xifaxan
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
XifaxanDrug
| Study group | Role | What they receive |
|---|---|---|
| Xifaxan | Experimental | Xifaxan 550 mg two times per day for three months Receives: Drug: Xifaxan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in alkaline phosphatase
An improvement in elevated levels of alkaline phosphatase to less than half of the initial level at study entry
Measured: Three months
Absence of treatment failure
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.
Measured: Three months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2012 (estimated)
- Study startAug 2012
- Primary completionDec 2013
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Sclerosing Cholangitis (PSC)Studies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jayant A. Talwalkar, Mayo Clinic
- Investigators
- Jayant Talwalkar, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01695174
- Sponsor's protocol ID
- 11-006516
- Acronym
- PSC
- Status last verified
- Oct 2014
- First submitted
- 25 Sep 2012
- Last updated
- 15 Oct 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 24914504
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Xifaxan in Patients With Primary Sclerosing Cholangitis
- ClinicalTrials.gov ID
- NCT01695174
- Acronym / sponsor ID
- PSC / 11-006516
- Conditions
- Primary Sclerosing Cholangitis (PSC)
- Keywords
- Primary Sclerosing Cholangitis, Xifaxan
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- Aug 2012
- Primary completion
- Dec 2013 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 27 Sep 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Xifaxan | Experimental | Drug: XifaxanXifaxan 550 mg two times per day for three months |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of PSC established by all of the following criteria:
- Both genders
- Adults: Ages 18-75 years.
- Pediatric: Weight > 40 kg
- Patient's informed consent for study participation
Key exclusion criteria (9 listed)
- 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
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 |
|---|---|---|
| Xifaxan | Drug | Xifaxan |
Primary outcome
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
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2014.
- Results publication: PubMed 24914504Tabibian 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.
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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 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 Xifaxan in people with Primary Sclerosing Cholangitis (PSC), 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 9 inclusion and 9 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 15 Oct 2014. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Improvement in alkaline phosphatase (time frame: Three months). The study lists 2 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 NCT01695174, 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 15 Oct 2014.