Trial Outcomes & Findings for Role of Exenatide in NASH-a Pilot Study (NCT NCT00650546)

Results Overview

Number of patients with liver histology improved with exenatide. The improvement of liver histology was defined as (1) no worsening of the fibrosis score, (11) improved score by at least one point in hepatocyte ballooning, and (111) either (a) improvement in NAS (NAFLD Activity Score) by two points spread across as least two of the three NAS components, or by (B)post-treatment NAS\<3.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

8 participants

Primary outcome timeframe

between baseline and 24-28 weeks after initiating treatment

Results posted on

2017-04-11

Participant Flow

2 recruitment sites: Department of Gastroenterology, San Antonio Uniformed Services Health Consortium, San Antonio, Texas, USA. Indiana University School of Medicine, Indianapolis, IN, USA

This was a single arm, open label study. Only study group received exenatide treatment. Eight adult patients with known type 2 DM and biopsy-proven NAFLD were treated with 5-10mcg subcutaneous exenatide for 28 week.All eight patients were started on exenatide 5 mcg injections twice a day and the dose was increased to 10 mcg injections twice a day

Participant milestones

Participant milestones
Measure	Exenatide Group Exenatide 5 micrograms SQ twice a day titrated to 10 mcg SQ twice a day as tolerated Exenatide : 5 mcg twice a day titrated to 10 mcg twice a day
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Role of Exenatide in NASH-a Pilot Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open-labeled Prospective Case Series n=8 Participants Exenatide 5 micrograms SQ twice a day titrated to 10 mcg SQ twice a day as tolerated Exenatide: 5 mcg twice a day titrated to 10 mcg twice a day
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	54 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Number of participants who had NAFLD Activity Score (NAS) pre/post 24-28 wks. after treatment	8 participants n=5 Participants
NAS score	4 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: between baseline and 24-28 weeks after initiating treatment

Number of patients with liver histology improved with exenatide. The improvement of liver histology was defined as (1) no worsening of the fibrosis score, (11) improved score by at least one point in hepatocyte ballooning, and (111) either (a) improvement in NAS (NAFLD Activity Score) by two points spread across as least two of the three NAS components, or by (B)post-treatment NAS\<3.

Outcome measures

Outcome measures
Measure	Treatment With Exenatide n=8 Participants eight adult patients with known type 2 DM and biopsy proven NAFLD
Number of Patients With Improvement in Liver Histology After Treatment With Exenatide	8 participants

PRIMARY outcome

Timeframe: Between baseline and 28 weeks of treatment with exenatide, sub q, 5-10 mcq.

The NAFLD Activity Score (NAS) is an underweight sum of steatosis (score 0-3), inflammation (score 0-3), ballooning scores (0-2). The NAS can range from 0-8 with the higher score indicating more aggressive disease.

Outcome measures

Outcome measures
Measure	Treatment With Exenatide n=8 Participants eight adult patients with known type 2 DM and biopsy proven NAFLD
Change in NAS	-1.5 units on a scale Standard Deviation 1.66

Adverse Events

Exenatide Group

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Exenatide Group n=8 participants at risk Exenatide 5 micrograms SQ twice a day titrated to 10 mcg SQ twice a day as tolerated Exenatide : 5 mcg twice a day titrated to 10 mcg twice a day
Gastrointestinal disorders abdominal pain along with nausea	25.0% 2/8 • Number of events 2 • 28 weeks

Additional Information

Patrick R Kenney, DO

Department of Gastroenterology, San Antonio Uniformed Services Health Consortium

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place