Pentoxifylline for Primary Biliary Cirrhosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01249092
Recruitment Status : Completed
First Posted : November 29, 2010
Results First Posted : December 9, 2013
Last Update Posted : December 9, 2013
Information provided by (Responsible Party):
Claudia Zein, MD, The Cleveland Clinic

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Primary Biliary Cirrhosis
Intervention: Drug: Pentoxifylline

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patient enrollment was initiated in December 2010 and a total of 20 patients were enrolled. The last patient to complete the pilot study completed participation in June 2012.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
This was a single arm open label pilot study where all subjects received the same intervention throughout the study duration. There were no different study groups and no randomization was involved.

Reporting Groups
Pentoxifylline 400 mg/d All patients received same intervention.

Participant Flow:   Overall Study
    Pentoxifylline 400 mg/d
Expected side effect of nausea/dyspepsia                1 
Unable to tolerate expected SE -nausea                1 

  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
Pentoxifylline 400 mg/d All patients received same intervention.

Baseline Measures
   Pentoxifylline 400 mg/d 
Overall Participants Analyzed 
[Units: Participants]
[Units: Participants]
<=18 years   0 
Between 18 and 65 years   6 
>=65 years   14 
[Units: Years]
Mean (Standard Deviation)
 57.7  (9.6) 
[Units: Participants]
Female   18 
Male   2 
Region of Enrollment 
[Units: Participants]
United States   20 

  Outcome Measures

1.  Primary:   Change in Serum Alkaline Phosphatase Levels.   [ Time Frame: 6 months ]

2.  Secondary:   Change in Serum Concentration of Tissue Inhibitor Metalloproteinase 1 (TIMP-1) After PTX Therapy.   [ Time Frame: 6 months ]

3.  Secondary:   Safety of Therapy in the Pilot Study of PTX Therapy in Patients With PBC Will be Assessed   [ Time Frame: 6 months ]

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Our study was a small open label pilot completed with no technical problems. The information generated will be useful in designing future larger studies. The main limitation is that PBC is a rare disease which resulted in prolonged enrollment phase.

  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact:  
Name/Title: Claudia O. Zein, MD
Organization: Cleveland Clinic
phone: 216-444-0421

Responsible Party: Claudia Zein, MD, The Cleveland Clinic Identifier: NCT01249092     History of Changes
Other Study ID Numbers: 07-1003
First Submitted: November 24, 2010
First Posted: November 29, 2010
Results First Submitted: July 31, 2013
Results First Posted: December 9, 2013
Last Update Posted: December 9, 2013