Trial Outcomes & Findings for Pentoxifylline for Primary Biliary Cirrhosis (NCT NCT01249092)

NCT ID: NCT01249092

Last Updated: 2013-12-09

Results Overview

Serum alkaline phosphatase levels at entry and at 6 months of therapy with PTX will be measured and compared.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 20 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2013-12-09

Participant Flow

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.

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.

Participant milestones

Measure	Pentoxifylline 400 mg/d All patients received same intervention.
Overall Study STARTED	20
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Pentoxifylline 400 mg/d All patients received same intervention.
Overall Study Expected side effect of nausea/dyspepsia	1
Overall Study Unable to tolerate expected SE -nausea	1

Baseline Characteristics

Pentoxifylline for Primary Biliary Cirrhosis

Baseline characteristics by cohort

Measure	Pentoxifylline 400 mg/d n=20 Participants All patients received same intervention.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	14 Participants n=5 Participants
Age Continuous	57.7 years STANDARD_DEVIATION 9.6 • n=5 Participants
Sex: Female, Male Female	18 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Serum alkaline phosphatase levels at entry and at 6 months of therapy with PTX will be measured and compared.

Outcome measures

Measure	Change in Alkaline Phosphatase After Pentoxifylline Therapy n=18 Participants
Change in Serum Alkaline Phosphatase Levels.	-57.3 U/L Standard Deviation 62.1

SECONDARY outcome

Timeframe: 6 months

Population: Serum samples from both timepoints (entry and end of study) were available in only 16 of the 18 subjects who completed the study.

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.

Outcome measures

Measure	Change in Alkaline Phosphatase After Pentoxifylline Therapy n=16 Participants
Change in Serum Concentration of Tissue Inhibitor Metalloproteinase 1 (TIMP-1) After PTX Therapy.	-5.69 ng/mL Standard Error 8.66

SECONDARY outcome

Timeframe: 6 months

The number of participants that experienced any severe adverse events will be monitored and recorded.

Outcome measures

Measure	Change in Alkaline Phosphatase After Pentoxifylline Therapy n=20 Participants
Safety of Therapy in the Pilot Study of PTX Therapy in Patients With PBC Will be Assessed	0 participants

Adverse Events

Pentoxifylline 400 mg/d

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Pentoxifylline 400 mg/d n=20 participants at risk All patients received same intervention.
Gastrointestinal disorders Nausea	35.0% 7/20 • Number of events 7 • 2 years and 6 months 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.
Gastrointestinal disorders Diarrhea	5.0% 1/20 • Number of events 1 • 2 years and 6 months 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.
Gastrointestinal disorders Dyspepsia	15.0% 3/20 • Number of events 3 • 2 years and 6 months 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.
Nervous system disorders Dizziness	5.0% 1/20 • Number of events 1 • 2 years and 6 months 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.

Additional Information

Claudia O. Zein, MD

Cleveland Clinic

Phone: 216-444-0421

Email: zeinc@ccf.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place