Trial Outcomes & Findings for Pentoxifylline/Nonalcoholic Steatohepatitis (NASH) Study: The Effect of Pentoxifylline on NASH (NCT NCT00267670)

NCT ID: NCT00267670

Last Updated: 2014-09-09

Results Overview

The primary goal of the study was to determine whether pentoxifylline (PTX) therapy improved serum ALT (\> or = 30% change from baseline to month 12) compared to placebo.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

26 participants

Primary outcome timeframe

baseline and 12 months

Results posted on

2014-09-09

Participant Flow

From March 2005 to March 2008 patients were recruited through the Northwestern Memorial Faculty Foundation Hepatology Clinic.

Subjects were excluded from the study if they had evidence of another form of liver disease or if they were HIV positive, pregnant or had evidence of ongoing alcohol consumption exceeding 20g(males) and 10g(females)daily. Furthermore, subjects were excluded if they were taking drugs known to cause steatohepatitis.

Participant milestones

Participant milestones
Measure
Pentoxifylline
This group was given pentoxifylline 400mg thrice daily (tid) for 1 year
Placebo
This group was given a capsule identical to pentoxifylline that contained sucrose.
Overall Study
STARTED
19
7
Overall Study
COMPLETED
15
7
Overall Study
NOT COMPLETED
4
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pentoxifylline/Nonalcoholic Steatohepatitis (NASH) Study: The Effect of Pentoxifylline on NASH

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Pentoxifylline
n=19 Participants
Patients in this group received pentoxifylline 400mg tid for 1 year.
Placebo
n=7 Participants
Patients in this group received a capsule identical to pentoxifylline that instead contained sucrose.
Total
n=26 Participants
Total of all reporting groups
Age, Categorical
<=18 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
19 Participants
n=5 Participants
7 Participants
n=7 Participants
26 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Age, Continuous
46 years
STANDARD_DEVIATION 10 • n=5 Participants
53 years
STANDARD_DEVIATION 8 • n=7 Participants
49.5 years
STANDARD_DEVIATION 10 • n=5 Participants
Sex: Female, Male
Female
12 Participants
n=5 Participants
3 Participants
n=7 Participants
15 Participants
n=5 Participants
Sex: Female, Male
Male
7 Participants
n=5 Participants
4 Participants
n=7 Participants
11 Participants
n=5 Participants
Region of Enrollment
United States
19 participants
n=5 Participants
7 participants
n=7 Participants
26 participants
n=5 Participants

PRIMARY outcome

Timeframe: baseline and 12 months

Population: The primary analysis was done as intention to treat. A secondary analysis was performed per protocol and there were no differences between the two analyses. Intention to treat results are reported.

The primary goal of the study was to determine whether pentoxifylline (PTX) therapy improved serum ALT (\> or = 30% change from baseline to month 12) compared to placebo.

Outcome measures

Outcome measures
Measure
Pentoxifylline
n=19 Participants
Patients in this group received pentoxifylline 400mg tid for 1 year.
Placebo
n=7 Participants
Patients in this group received a capsule identical to pentoxifylline that instead contained sucrose.
The Number of Participants With a 30% Reduction in Alanine Aminotransferase (ALT) Treated With Pentoxifylline (PTX) or Placebo for 12 Months.
19 participants
7 participants

SECONDARY outcome

Timeframe: one year

Population: Intention to Treat with last observation carried forward

The mean change from baseline to month 12 in proinflammatory cytokines (such as TNF-α) and gene expresssion were the secondary endpoints and were analyzed with the same analysis of covariance model and summary statistics specified for the primary endpoint. Differences were regarded as statistically significant when P \< 0.05. The results for TNF-α are reported here. Interleukin-6 \[IL-6\], IL-10) and expression of TNF-alpha Receptors (p55 and p75) had insufficient data for statistical analysis.

Outcome measures

Outcome measures
Measure
Pentoxifylline
n=19 Participants
Patients in this group received pentoxifylline 400mg tid for 1 year.
Placebo
n=7 Participants
Patients in this group received a capsule identical to pentoxifylline that instead contained sucrose.
The Effect of Pentoxifylline on Change in Tumor Necrosis Factor [TNF]-α Levels in Patients With NASH
-117.9 pg/dL
Standard Error 109.5
18.3 pg/dL
Standard Error 64.4

SECONDARY outcome

Timeframe: baseline and one year

Values represent changes in leptin from baseline to 12 months in patients treated with pentoxifylline or placebo.

Outcome measures

Outcome measures
Measure
Pentoxifylline
n=19 Participants
Patients in this group received pentoxifylline 400mg tid for 1 year.
Placebo
n=7 Participants
Patients in this group received a capsule identical to pentoxifylline that instead contained sucrose.
Change in Serum Leptin Levels in Patients Treated With Pentoxifylline or Placebo for 12 Months
0.78 ng/mL
Standard Error 0.07
0.78 ng/mL
Standard Error 0.08

SECONDARY outcome

Timeframe: one year

Outcome measures

Outcome measures
Measure
Pentoxifylline
n=19 Participants
Patients in this group received pentoxifylline 400mg tid for 1 year.
Placebo
n=7 Participants
Patients in this group received a capsule identical to pentoxifylline that instead contained sucrose.
Change in Serum Adiponectin Levels in Patients Treated With Pentoxifylline or Placebo for 12 Months
0.4 ug/mL
Standard Error 0.3
0.8 ug/mL
Standard Error 0.4

Adverse Events

Pentoxifylline

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Mary E. Rinella, MD

Northwestern University

Phone: 312-503-4592

Results disclosure agreements

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