Trial Outcomes & Findings for Pentoxifylline in Patients With Nonalcoholic Steatohepatitis (NCT NCT00590161)
NCT ID: NCT00590161
Last Updated: 2013-09-26
Results Overview
The NAFLD Activity Score (NAS) grades NAFLD on liver biopsy based on the individual scoring of steatosis, inflammation and balloning. The NAS is assessed on a scale of 0 to 8 with higher scores indicating more severe disease and lower scores indicating less severe disease. NAS is obtained by adding steatosis(assessed on a scale of 0 to 3), inflammation (assessed on a scale of 0 to 3) and ballooning (assessed on a scale of 0 to 2).
COMPLETED
PHASE2
55 participants
1 year (Baseline liver biopsy done at study entry, and subsequent liver biopsy done after one year of therapy with pentoxifylline or placebo)
2013-09-26
Participant Flow
Patients were recruited between December 2006 and February 2009 at the Liver Clinics of both participating institutions.
Patients were randomized in a double-blind fashion. Double blinding was maintained until study completion by all subjects.
Participant milestones
| Measure |
Pentoxifylline (PTX) 400 mg PO Three Times Daily (TID)
26 subjects received PTX at dose above for one year
|
Placebo TID
29 subjects received placebo as above for one year
|
|---|---|---|
|
Overall Study
STARTED
|
26
|
29
|
|
Overall Study
COMPLETED
|
23
|
26
|
|
Overall Study
NOT COMPLETED
|
3
|
3
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pentoxifylline in Patients With Nonalcoholic Steatohepatitis
Baseline characteristics by cohort
| Measure |
Pentoxifylline (PTX) 400 mg PO (by Mouth) TID
n=26 Participants
26 subjects received PTX at dose above for one year
|
Placebo TID
n=29 Participants
29 subjects received placebo as above for one year
|
Total
n=55 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
23 Participants
n=93 Participants
|
27 Participants
n=4 Participants
|
50 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
5 Participants
n=27 Participants
|
|
Age Continuous
|
50.5 years
STANDARD_DEVIATION 12.7 • n=93 Participants
|
49.6 years
STANDARD_DEVIATION 9.6 • n=4 Participants
|
50 years
STANDARD_DEVIATION 11.1 • n=27 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=93 Participants
|
9 Participants
n=4 Participants
|
17 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=93 Participants
|
20 Participants
n=4 Participants
|
38 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
26 participants
n=93 Participants
|
29 participants
n=4 Participants
|
55 participants
n=27 Participants
|
PRIMARY outcome
Timeframe: 1 year (Baseline liver biopsy done at study entry, and subsequent liver biopsy done after one year of therapy with pentoxifylline or placebo)Population: Intention to treat analysis included all participants and for this analysis patients without available end of study liver biopsy were imputed as treatment failures. Results showed here are continuous variable analysis of NAS score change in patients with available end of study liver biopsy (per protocol analysis)
The NAFLD Activity Score (NAS) grades NAFLD on liver biopsy based on the individual scoring of steatosis, inflammation and balloning. The NAS is assessed on a scale of 0 to 8 with higher scores indicating more severe disease and lower scores indicating less severe disease. NAS is obtained by adding steatosis(assessed on a scale of 0 to 3), inflammation (assessed on a scale of 0 to 3) and ballooning (assessed on a scale of 0 to 2).
Outcome measures
| Measure |
Pentoxifylline 400 mg PO Tid
n=20 Participants
26 subjects received PTX at dose above for one year
|
Placebo Tid
n=26 Participants
29 subjects received placebo as above for one year
|
|---|---|---|
|
Histological Improvement of at Least 2 Points in NAFLD Activity Score (NAS) on Liver Biopsy After One Year.
|
-1.6 NAS score units
Standard Deviation 1.1
|
-0.1 NAS score units
Standard Deviation 1.4
|
Adverse Events
Pentoxifylline 400 mg PO Tid
Placebo Tid
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Pentoxifylline 400 mg PO Tid
n=26 participants at risk
26 subjects received PTX at dose above for one year
|
Placebo Tid
n=29 participants at risk
29 subjects received placebo as above for one year
|
|---|---|---|
|
Gastrointestinal disorders
Nausea
|
23.1%
6/26 • Number of events 6
|
13.8%
4/29 • Number of events 4
|
|
Gastrointestinal disorders
Bloating
|
7.7%
2/26 • Number of events 2
|
10.3%
3/29 • Number of events 3
|
|
General disorders
Headache
|
7.7%
2/26 • Number of events 2
|
3.4%
1/29 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/26
|
6.9%
2/29 • Number of events 2
|
|
General disorders
Lightheadedness
|
3.8%
1/26 • Number of events 1
|
10.3%
3/29 • Number of events 3
|
|
Gastrointestinal disorders
Diarrhea
|
3.8%
1/26 • Number of events 1
|
10.3%
3/29 • Number of events 3
|
|
General disorders
Chest pain
|
0.00%
0/26
|
6.9%
2/29 • Number of events 2
|
|
General disorders
Fatigue
|
0.00%
0/26
|
6.9%
2/29 • Number of events 2
|
Additional Information
Claudia O. Zein, MD, MSc
Cleveland Clinic Lerner College of Medicine of Case Western Reserve University
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place