Trial Outcomes & Findings for Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome (NCT NCT01444898)

NCT ID: NCT01444898

Last Updated: 2016-09-29

Results Overview

Change in weight (kg) after 6 months of treatment with study drug. Described as mean +/- SD

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 10 participants
• Primary outcome timeframe: 6 months
• Results posted on: 2016-09-29

Participant Flow

Participant milestones

Measure	Exenatide All subjects enrolled in this study will be given Exenatide for 6 months. The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).
Overall Study STARTED	10
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Exenatide All subjects enrolled in this study will be given Exenatide for 6 months. The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome

Baseline characteristics by cohort

Measure	Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).
Age, Categorical <=18 years	6 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	16 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants
Weight	102.5 kg STANDARD_DEVIATION 18.6 • n=5 Participants
BMI	41.7 kg m^-2 n=5 Participants
BMI Z-Score	3.5 units on a scale STANDARD_DEVIATION 1.1 • n=5 Participants
Glycosylated hemoglobin (HbA1c)	5.9 percentage of total hemoglobin n=5 Participants
Insulin	10.5 uIU ml^-1 n=5 Participants
Leptin	36.4 ng ml ^-1 STANDARD_DEVIATION 18.3 • n=5 Participants
Acy Ghr	362.3 pg ml ^-1 n=5 Participants
PP	89 pg ml ^-1 n=5 Participants
Appetite Total Appetite Score	32.2 units on a scale STANDARD_DEVIATION 8.7 • n=5 Participants
Appetite Appetite Behavior	14.5 units on a scale STANDARD_DEVIATION 4.9 • n=5 Participants
Appetite Appetite Drive	12.6 units on a scale STANDARD_DEVIATION 3.1 • n=5 Participants
Appetite Appetite Severity	5.1 units on a scale STANDARD_DEVIATION 1.5 • n=5 Participants
Adiposity	20 kg STANDARD_DEVIATION 4.6 • n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Change in weight (kg) after 6 months of treatment with study drug. Described as mean +/- SD

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in Weight

-.5 kg Standard Deviation .9

PRIMARY outcome

Timeframe: 6 months

Prior to analysis, distributions were evaluated for normality and natural log transformation was performed to analyse data not normally distributed. Data are presented as mean ±SD unless not normally distributed, in which case they are presented as median with intra-quartile ranges (25th and 75th percentiles). Within-subject changes between visits were analysed by mixed model repeated measures. When the overall F-test for difference among visits was significant, Dunnett-adjusted pairwise comparisons were made between baseline and each subsequent visit.

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

% Change in Body Mass Index (BMI)

1.3 % change in BMI Standard Deviation 2

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in BMI Z-Score

.1 units on a scale Standard Deviation .4

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in HbA1c (%)

-.3 percentage Standard Deviation .2

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in Insulin Levels

3 u/U ml^-1 Standard Deviation .5

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in Leptin

-7.4 ng ml^-1 Standard Deviation 3.8

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in Acy Ghr

263 pg ml^-1 Standard Deviation 99.5

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Measure

Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Change in Pancreatic Peptide (PP)

15 pg ml^-1 Standard Deviation 6

PRIMARY outcome

Timeframe: 6 months

Appetite scores using a syndrome-validated hyperphagia questionnaire

11 item questionnaire divided into subcategories of behavior (5 questions), drive (4 questions), severity (2 questions). Tallied and analyzed as total and subcategory scores. Each question scored 1-5 with higher scores correlating with worse hyperphagia.

Possible ranges: Total 11-55, behavior 5-25, drive 4-20, severity 2-10

Outcome measures

Measure	Exenatide n=10 Participants All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).
Appetite Scores Total Appetite Score Baseline	32.2 units on a scale Standard Deviation 8.7
Appetite Scores Total Appetite Score 6 month	25.4 units on a scale Standard Deviation 7.2
Appetite Scores Behavior Score Baseline	14.5 units on a scale Standard Deviation 4.9
Appetite Scores Behavior Score 6 months	10.6 units on a scale Standard Deviation 3.8
Appetite Scores Drive Score Baseline	12.6 units on a scale Standard Deviation 3.1
Appetite Scores Drive Score 6 months	10.4 units on a scale Standard Deviation 2.7
Appetite Scores Severity Score Baseline	5.1 units on a scale Standard Deviation 1.5
Appetite Scores Severity Score 6 months	4.4 units on a scale Standard Deviation 2.2

Adverse Events

Exenatide

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure

Exenatide n=10 participants at risk All subjects enrolled in this study will be given Exenatide for 6 months. Exenatide: The investigators will give patients naive to GLP-1 agonists exenatide per manufacturer dosing recommendations for 6 months. The investigators will begin by giving 5 mcg subcutaneously twice a day for 1 month and then increase the dose to 10 mcg subcutaneously twice a day for the remainder of the study (5 months).

Gastrointestinal disorders abdominal discomfort with diarrhea

20.0% 2/10 • Number of events 2

Additional Information

Dr. Mitchell Geffner

Children's Hospital Los Angeles

Phone: 323-361-7032

Email: mgeffner@chla.usc.edu

Results disclosure agreements

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