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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.
 
ClinicalTrials.gov Identifier: NCT01249092
Recruitment Status : Completed
First Posted : November 29, 2010
Results First Posted : December 9, 2013
Last Update Posted : December 9, 2013
Sponsor:
Information provided by (Responsible Party):
Claudia Zein, MD, The Cleveland Clinic

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Primary Biliary Cirrhosis
Intervention Drug: Pentoxifylline
Enrollment 20
Recruitment Details 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 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.
Arm/Group Title Pentoxifylline 400 mg/d
Hide Arm/Group Description All patients received same intervention.
Period Title: Overall Study
Started 20
Completed 18
Not Completed 2
Reason Not Completed
Expected side effect of nausea/dyspepsia             1
Unable to tolerate expected SE -nausea             1
Arm/Group Title Pentoxifylline 400 mg/d
Hide Arm/Group Description All patients received same intervention.
Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
<=18 years
0
   0.0%
Between 18 and 65 years
6
  30.0%
>=65 years
14
  70.0%
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 20 participants
57.7  (9.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Female
18
  90.0%
Male
2
  10.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 20 participants
20
1.Primary Outcome
Title Change in Serum Alkaline Phosphatase Levels.
Hide Description Serum alkaline phosphatase levels at entry and at 6 months of therapy with PTX will be measured and compared.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Change in Alkaline Phosphatase After Pentoxifylline Therapy
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 18
Mean (Standard Deviation)
Unit of Measure: U/L
-57.3  (62.1)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Change in Alkaline Phosphatase After Pentoxifylline Therapy
Comments A p-value < 0.05 was considered statistically significant and all analyses were carried out using SAS version 9.2 (The SAS Institute, Cary, NC). Matched pairs t-test was used to compare the change in alkaline phosphatase from baseline. The efficacy of therapy was measured based on improvement in AP levels after therapy with PTX. AP levels at end of the study were compared with values at baseline by matched pairs t-test.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.001
Comments This study tested the hypothesis that treatment with pentoxifylline would result in a statistically significant change from baseline in the level of alkaline phosphatase.
Method Paired t-test
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value -57.3
Confidence Interval (2-Sided) 95%
-88.2 to -26.4
Parameter Dispersion
Type: Standard Deviation
Value: 62.1
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Change in Serum Concentration of Tissue Inhibitor Metalloproteinase 1 (TIMP-1) After PTX Therapy.
Hide Description 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.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Serum samples from both timepoints (entry and end of study) were available in only 16 of the 18 subjects who completed the study.
Arm/Group Title Change in Serum TIMP-1 (Tissue Inhibitor metalloproteinase1)
Hide Arm/Group Description:
[Not Specified]
Overall Number of Participants Analyzed 16
Mean (Standard Error)
Unit of Measure: ng/mL
-5.69  (8.66)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Change in Serum TIMP-1 (Tissue Inhibitor metalloproteinase1)
Comments Change from baseline in TIMP-1 levels was assessed by paired-t test. Distribution the variable values was assessed using normal probability plots. Matched pairs t-test was used to compare the change from baseline.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5
Comments Hypothesis tested if TIMP-1 levels after therapy with pentoxifylline changed significantly from baseline from baseline.
Method Paired t-test.
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -5.69
Confidence Interval (2-Sided) 95%
-24.14 to 8.68
Parameter Dispersion
Type: Standard Error of the mean
Value: 8.66
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Safety of Therapy in the Pilot Study of PTX Therapy in Patients With PBC Will be Assessed
Hide Description The number of participants that experienced any severe adverse events will be monitored and recorded.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pentoxifylline 400 mg/d
Hide Arm/Group Description:
Descriptive information only of small open label pilot. Small patient number not amenable for any valid statistical comparisons regarding side effects. All patients received same intervention. No SAEs occurred.
Overall Number of Participants Analyzed 20
Measure Type: Number
Unit of Measure: participants
0
Time Frame 2 years and 6 months
Adverse Event Reporting Description Patients were followed for adverse events and side effect through the duration of the study and on subsequent follow up. Patients were contacted directly by PI and questionnaire about adverse effects was completed at weeks 4, 8, 12, 16, 20, and 24 during the study and then subsequently on follow up.
 
Arm/Group Title Pentoxifylline 400 mg/d
Hide Arm/Group Description All patients received same intervention.
All-Cause Mortality
Pentoxifylline 400 mg/d
Affected / at Risk (%)
Total   --/--    
Hide Serious Adverse Events
Pentoxifylline 400 mg/d
Affected / at Risk (%) # Events
Total   0/20 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Pentoxifylline 400 mg/d
Affected / at Risk (%) # Events
Total   8/20 (40.00%)    
Gastrointestinal disorders   
Nausea  1 [1]  7/20 (35.00%)  7
Diarrhea  1  1/20 (5.00%)  1
Dyspepsia  1  3/20 (15.00%)  3
Nervous system disorders   
Dizziness  1  1/20 (5.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (3.0)
[1]
Nausea rated more than mild temporary nausea (mild temporary nausea commonly occurs for a few days when initiating pentoxifylline)
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.
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
Layout table for Results Point of Contact information
Name/Title: Claudia O. Zein, MD
Organization: Cleveland Clinic
Phone: 216-444-0421
EMail: zeinc@ccf.org
Layout table for additonal information
Responsible Party: Claudia Zein, MD, The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT01249092    
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