Trial Outcomes & Findings for Pioglitazone vs Vitamin E vs Placebo for Treatment of Non-Diabetic Patients With Nonalcoholic Steatohepatitis (PIVENS) (NCT NCT00063622)

NCT ID: NCT00063622

Last Updated: 2018-04-06

Results Overview

Total nonalcoholic fatty liver disease (NAFLD) activity was assessed on a scale of 0 to 8, with higher scores indicating more severe disease; the components of this measure include steatosis (assessed on a scale of 0 to 3), lobular inflammation (assessed on a scale of 0 to 3), and hepatocellular ballooning (assessed on a scale of 0 to 2). The primary outcome was an improvement in histological findings from baseline to 96 weeks, which required an improvement by 1 or more points in the hepatocellular ballooning score; no increase in the fibrosis score; and either a decrease in the activity score for nonalcoholic fatty liver disease to a score of 3 or less or a decrease in the activity score of at least 2 points, with at least a 1-point decrease in either the lobular inflammation or steatosis score.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

247 participants

Primary outcome timeframe

baseline and 96 weeks

Results posted on

2018-04-06

Participant Flow

PIVENS enrollment started in January 2005 and ended in January 2007.

A total of 339 patients were registered and screened for PIVENS trial, 92 of whom (27%) were found ineligible. The failure to meet histological entry criteria and fasting blood glucose \>125 mg/dL were the most frequent reasons for ineligibility.

Participant milestones

Participant milestones
Measure
Pioglitazone
Pioglitazone at a dose of 30 mg daily
Vitamin E
Vitamin E at a dose of 800 IU daily
Placebo
Placebo Pioglitazone and Placebo Vitamin E
Overall Study
STARTED
80
84
83
Overall Study
COMPLETED
70
80
72
Overall Study
NOT COMPLETED
10
4
11

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pioglitazone vs Vitamin E vs Placebo for Treatment of Non-Diabetic Patients With Nonalcoholic Steatohepatitis (PIVENS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Pioglitazone
n=80 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=84 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=83 Participants
Placebo Pioglitazone and Placebo Vitamin E
Total
n=247 Participants
Total of all reporting groups
Age, Continuous
47.0 years
STANDARD_DEVIATION 12.6 • n=99 Participants
46.6 years
STANDARD_DEVIATION 12.1 • n=107 Participants
45.4 years
STANDARD_DEVIATION 11.2 • n=206 Participants
46.3 years
STANDARD_DEVIATION 11.9 • n=7 Participants
Sex: Female, Male
Female
47 Participants
n=99 Participants
52 Participants
n=107 Participants
48 Participants
n=206 Participants
147 Participants
n=7 Participants
Sex: Female, Male
Male
33 Participants
n=99 Participants
32 Participants
n=107 Participants
35 Participants
n=206 Participants
100 Participants
n=7 Participants
Region of Enrollment
United States
80 participants
n=99 Participants
84 participants
n=107 Participants
83 participants
n=206 Participants
247 participants
n=7 Participants
Total nonalcoholic fatty liver disease (NAFLD) activity score
5.0 scores on a scale
STANDARD_DEVIATION 1.4 • n=99 Participants
5.1 scores on a scale
STANDARD_DEVIATION 1.4 • n=107 Participants
4.8 scores on a scale
STANDARD_DEVIATION 1.4 • n=206 Participants
4.9 scores on a scale
STANDARD_DEVIATION 1.4 • n=7 Participants

PRIMARY outcome

Timeframe: baseline and 96 weeks

Population: All randomized participants were included in the analysis of the primary outcome.

Total nonalcoholic fatty liver disease (NAFLD) activity was assessed on a scale of 0 to 8, with higher scores indicating more severe disease; the components of this measure include steatosis (assessed on a scale of 0 to 3), lobular inflammation (assessed on a scale of 0 to 3), and hepatocellular ballooning (assessed on a scale of 0 to 2). The primary outcome was an improvement in histological findings from baseline to 96 weeks, which required an improvement by 1 or more points in the hepatocellular ballooning score; no increase in the fibrosis score; and either a decrease in the activity score for nonalcoholic fatty liver disease to a score of 3 or less or a decrease in the activity score of at least 2 points, with at least a 1-point decrease in either the lobular inflammation or steatosis score.

Outcome measures

Outcome measures
Measure
Pioglitazone
n=80 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=84 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=83 Participants
Placebo Pioglitazone and Placebo Vitamin E
Number of Participants With Improvement in Non-alcoholic Fatty Liver Disease (NAFLD) Activity Defined by Change in Standardized Scoring of Liver Biopsies at Baseline and After 96 Weeks of Treatment.
27 participants
36 participants
16 participants

SECONDARY outcome

Timeframe: baseline and 96 weeks

Population: Number of subjects with biopsy specimens at baseline and 96 weeks

Steatosis is assessed on a scale of 0 to 3 with higher scores indicating more severe steatosis. This secondary outcome measure is the number of participants that experienced a decrease in steatosis score, which indicates improvement in steatosis.

Outcome measures

Outcome measures
Measure
Pioglitazone
n=70 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=80 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=72 Participants
Placebo Pioglitazone and Placebo Vitamin E
Number of Participants With Improvement in Steatosis
48 participants
43 participants
22 participants

SECONDARY outcome

Timeframe: baseline and 96 weeks

Population: Number of subjects with biopsy specimens at baseline and 96 weeks

Lobular inflammation is assessed on a scale of 0 to 3 with higher scores indicating more severe lobular inflammation. This secondary outcome measure is the number of participants that experienced a decrease in lobular inflammation score, which indicates improvement in lobular inflammation.

Outcome measures

Outcome measures
Measure
Pioglitazone
n=70 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=80 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=72 Participants
Placebo Pioglitazone and Placebo Vitamin E
Number of Participants With Improvement in Lobular Inflammation
41 participants
43 participants
25 participants

SECONDARY outcome

Timeframe: baseline and 96 weeks

Population: Number of subjects with biopsy specimens at baseline and 96 weeks

Hepatocellular ballooning is assessed on a scale of 0 to 2 with higher scores indicating more severe hepatocellular ballooning. This secondary outcome measure is the number of participants that experienced a decrease in hepatocellular ballooning score, which indicates improvement in hepatocellular ballooning.

Outcome measures

Outcome measures
Measure
Pioglitazone
n=70 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=80 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=72 Participants
Placebo Pioglitazone and Placebo Vitamin E
Number of Participants With Improvement in Hepatocellular Ballooning
31 participants
40 participants
21 participants

SECONDARY outcome

Timeframe: baseline and 96 weeks

Population: Number of subjects with biopsy specimens at baseline and 96 weeks

Fibrosis is assessed on a scale of 0 to 4 with higher scores indicating more severe fibrosis. This secondary outcome measure is the number of participants that experienced a decrease in fibrosis score, which indicates improvement in fibrosis.

Outcome measures

Outcome measures
Measure
Pioglitazone
n=70 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=80 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=72 Participants
Placebo Pioglitazone and Placebo Vitamin E
Number of Participants With Improvement in Fibrosis
31 participants
33 participants
22 participants

SECONDARY outcome

Timeframe: baseline and 96 weeks

Population: Number of subjects with biopsy specimens at baseline and 96 weeks

The criteria for nonalcoholic steatohepatitis was definite or possible steatohepatitis (assessed by a pathologist) with an activity score of 5 or more, or definite steatohepatitis (confirmed by two pathologists) with an activity score of 4. This secondary outcome measure is the number of participants who met this definition at baseline and did not meet this definition after 96 weeks of treatment and thus had a resolution of steatohepatitis.

Outcome measures

Outcome measures
Measure
Pioglitazone
n=70 Participants
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=80 Participants
Vitamin E at a dose of 800 IU daily
Placebo
n=72 Participants
Placebo Pioglitazone and Placebo Vitamin E
Number of Participants With Resolution of Definite Nonalcoholic Steatohepatitis
33 participants
29 participants
15 participants

Adverse Events

Pioglitazone

Serious events: 2 serious events
Other events: 41 other events
Deaths: 0 deaths

Vitamin E

Serious events: 7 serious events
Other events: 46 other events
Deaths: 0 deaths

Placebo

Serious events: 10 serious events
Other events: 40 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Pioglitazone
n=80 participants at risk
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=84 participants at risk
Vitamin E at a dose of 800 IU daily
Placebo
n=83 participants at risk
Placebo Pioglitazone and Placebo Vitamin E
General disorders
Severe adverse event
2.5%
2/80
8.3%
7/84
12.0%
10/83

Other adverse events

Other adverse events
Measure
Pioglitazone
n=80 participants at risk
Pioglitazone at a dose of 30 mg daily
Vitamin E
n=84 participants at risk
Vitamin E at a dose of 800 IU daily
Placebo
n=83 participants at risk
Placebo Pioglitazone and Placebo Vitamin E
Cardiac disorders
Cardiovascular Event
12.5%
10/80
14.3%
12/84
14.5%
12/83
Musculoskeletal and connective tissue disorders
Bone fracture
3.8%
3/80
3.6%
3/84
6.0%
5/83
Endocrine disorders
Diabetes
0.00%
0/80
4.8%
4/84
0.00%
0/83
Hepatobiliary disorders
Cirrhosis
0.00%
0/80
1.2%
1/84
0.00%
0/83
General disorders
Other mild and moderate adverse event
35.0%
28/80
31.0%
26/84
27.7%
23/83

Additional Information

Arun J. Sanyal

Virginia Commonwealth University

Results disclosure agreements

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