Primary Biliary CirrhosisPhase 2NCT01249092
Pentoxifylline for Primary Biliary Cirrhosis
A Phase 2 study testing Pentoxifylline in people with Primary Biliary Cirrhosis, run by The Cleveland Clinic at 1 site in United States.
Trial summary
Primary biliary cirrhosis (PBC) is cholestatic liver disease characterized by progressive destruction of small bile ducts within the liver that can lead to end stage liver disease and all its complications. Read more about this study
- Who is it for?
- Ages 18 to 76 years
- Conditions the study is for
- Primary Biliary Cirrhosis
- What is being tested?
- Pentoxifylline
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2010to Mar 2013
- Phase
- Phase 2
- 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 and female patients ages 18 to 76 years.
- Established diagnosis of PBC based on at least three of the following criteria:
- Detectable anti-mitochondrial antibodies (AMA)
- Cholestatic biochemical pattern
- Liver biopsy compatible with PBC
- Appropriate exclusion of other liver diseases.
- Therapy with UDCA at adequate dose (13-15mg/kg/d) for at least six months and evidence of suboptimal response defined by alkaline phosphatase levels that did not normalize and remain elevated by at least 1.5 times the upper limit of normal.
- No history or present hepatic decompensation (e.g. variceal hemorrhage, encephalopathy, or poorly controlled ascites).
Who cannot join
- Findings highly suggestive of liver disease of other etiology.
- A score >=10 points on the Revised Scoring System for autoimmune hepatitis (AIH), supporting a diagnosis of PBC/AIH overlap.
- Patients on steroids (systemic), immunosuppressants, or immunomodulatory agents within the previous 6 months.
- Patients with clinical or laboratory evidence suggestive of decompensated cirrhosis.
- Hypersensitivity to PTX or the methylxanthines (caffeine, theophylline, theobromine).
- History of cerebral or retinal hemorrhage.
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- Other medical comorbidities (such as cardiac, renal, cancer) that would interfere with completion of the study.
- Patients taking Theophylline or Coumadin because of potential drug-drug interactions with PTX. In addition, patients taking low molecular weight heparin preparations.
- Pregnant or nursing women.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study of Pentoxifylline for the Treatment of Primary Biliary Cirrhosis
Primary biliary cirrhosis (PBC) is cholestatic liver disease characterized by progressive destruction of small bile ducts within the liver that can lead to end stage liver disease and all its complications.
Although ursodeoxycholic acid (UDCA) is associated with increased survival in many patients with PBC, there is absence of an adequate response to UDCA in a significant proportion of PBC patients.
Read more
Tumor necrosis factor alpha (TNF-alpha) is a cytokine that plays an important role in the pathogenesis of PBC. Other fibrosis biomarkers such as tissue metallo proteinase 1 (TIMP-1) are associated with progression of liver fibrosis in PBC. Pentoxifylline (PTX) is a methylxanthine derivative that inhibits pro-inflammatory cytokines and also has shown anti-fibrotic effects in serum of patients with PBC. Furthermore, PTX has well known clinical and safety profiles. The main hypothesis of this study is that therapy with pentoxifylline (PTX) will result in improvement of liver disease in PBC patients who are incomplete responders to UDCA.
The focus of this proposal is on the effectiveness of PTX in improving laboratory parameters of liver disease and levels of cytokines involved in the pathogenesis of the disease in patients with PBC.
- Conditions
- Primary Biliary Cirrhosis
- Being tested
- Pentoxifylline
- Keywords
- biliary cirrhosis
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PentoxifyllineDrug
Patients will take 400mg of pentoxifylline three times daily for a total duration of 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Pentoxifylline 400 mg TID | Experimental | This study is an open label pilot with only one arm. Receives: Drug: Pentoxifylline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Serum Alkaline Phosphatase Levels.
Serum alkaline phosphatase levels at entry and at 6 months of therapy with PTX will be measured and compared.
Measured: 6 months
Secondary outcomes (2)
Change in Serum Concentration of Tissue Inhibitor Metalloproteinase 1 (TIMP-1) After PTX Therapy.
Serum concentration of tissue inhibitor metalloproteinase 1 (TIMP-1), a fibrosis biomarker of interest, will be measured and the change in serum levels between entry and end of study will be calculated.
Measured: 6 months
Safety of Therapy in the Pilot Study of PTX Therapy in Patients With PBC Will be Assessed
The number of participants that experienced any severe adverse events will be monitored and recorded.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2010 (estimated)
- Study startNov 2010
- Primary completionJun 2012
- Study completionMar 2013
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.
- Other trials for Primary Biliary CirrhosisStudies of the same condition, open and under way.
- More trials run by The Cleveland 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Cleveland Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Claudia Zein, MD, The Cleveland Clinic
- Investigators
- Claudia O. Zein, MD, MSc, Principal investigator, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01249092
- Sponsor's protocol ID
- 07-1003
- Status last verified
- Oct 2013
- First submitted
- 24 Nov 2010
- Last updated
- 9 Dec 2013
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Pentoxifylline for the Treatment of Primary Biliary Cirrhosis
- ClinicalTrials.gov ID
- NCT01249092
- Acronym / sponsor ID
- 07-1003
- Conditions
- Primary Biliary Cirrhosis
- Keywords
- biliary cirrhosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- The Cleveland ClinicUniversity, hospital or other
- Study start
- Nov 2010
- Primary completion
- Jun 2012 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 29 Nov 2010 (estimated)
- Results
- Posted 9 Dec 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary biliary cirrhosis (PBC) is cholestatic liver disease characterized by progressive destruction of small bile ducts within the liver that can lead to end stage liver disease and all its complications.
Although ursodeoxycholic acid (UDCA) is associated with increased survival in many patients with PBC, there is absence of an adequate response to UDCA in a significant proportion of PBC patients.
Tumor necrosis factor alpha (TNF-alpha) is a cytokine that plays an important role in the pathogenesis of PBC. Other fibrosis biomarkers such as tissue metallo proteinase 1 (TIMP-1) are associated with progression of liver fibrosis in PBC. Pentoxifylline (PTX) is a methylxanthine derivative that inhibits pro-inflammatory cytokines and also has shown anti-fibrotic effects in serum of patients with PBC. Furthermore, PTX has well known clinical and safety profiles. The main hypothesis of this study is that therapy with pentoxifylline (PTX) will result in improvement of liver disease in PBC patients who are incomplete responders to UDCA.
The focus of this proposal is on the effectiveness of PTX in improving laboratory parameters of liver disease and levels of cytokines involved in the pathogenesis of the disease in patients with PBC.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pentoxifylline 400 mg TID | Experimental | Drug: PentoxifyllineThis study is an open label pilot with only one arm. |
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 and female patients ages 18 to 76 years.
- Established diagnosis of PBC based on at least three of the following criteria:
- Therapy with UDCA at adequate dose (13-15mg/kg/d) for at least six months and evidence of suboptimal response defined by alkaline phosphatase levels that did not normalize and remain elevated by at least 1.5 times the upper limit of normal.
- No history or present hepatic decompensation (e.g. variceal hemorrhage, encephalopathy, or poorly controlled ascites).
Key exclusion criteria (9 listed)
- Findings highly suggestive of liver disease of other etiology.
- A score >=10 points on the Revised Scoring System for autoimmune hepatitis (AIH), supporting a diagnosis of PBC/AIH overlap.
- Patients on steroids (systemic), immunosuppressants, or immunomodulatory agents within the previous 6 months.
- Patients with clinical or laboratory evidence suggestive of decompensated cirrhosis.
- Hypersensitivity to PTX or the methylxanthines (caffeine, theophylline, theobromine).
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 |
|---|---|---|
| PentoxifyllinePatients will take 400mg of pentoxifylline three times daily for a total duration of 6 months. | Drug | Pentoxifylline 400 mg TID |
Primary outcome
Change in Serum Alkaline Phosphatase Levels.
Time frame: 6 months
Serum alkaline phosphatase levels at entry and at 6 months of therapy with PTX will be measured and compared.
2 secondary outcomes
Change in Serum Concentration of Tissue Inhibitor Metalloproteinase 1 (TIMP-1) After PTX Therapy.
Time frame: 6 months
Safety of Therapy in the Pilot Study of PTX Therapy in Patients With PBC Will be Assessed
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Dec 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Claudia O. Zein, MD, MSc principal investigator
The Cleveland Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pentoxifylline in people with Primary Biliary Cirrhosis, run by The Cleveland 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 8 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 9 Dec 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 9 Dec 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 in Serum Alkaline Phosphatase Levels. (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Dec 2013; AzTrials last refreshed its copy on 28 Oct 2026.