Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome
|ClinicalTrials.gov Identifier: NCT01444898|
Recruitment Status : Completed
First Posted : October 3, 2011
Results First Posted : September 29, 2016
Last Update Posted : September 29, 2016
|Condition or disease||Intervention/treatment||Phase|
|Prader-Willi Syndrome||Drug: Exenatide||Not Applicable|
Prader-Willi syndrome (PWS) is associated with hyperphagia and hyperghrelinemia with major morbidity because of obesity without effective medical treatment targeting hyperphagia. Exenatide (Byetta [synthetic Exendin-4]; AstraZeneca, Wilmington DE) is a GLP-1 receptor agonist which reduces appetite and weight and may be an effective treatment in PWS.
OBJECTIVE: The objective of this study is to determine the effect of a 6-month trial of exenatide on appetite, weight and gut hormones in youth with PWS.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Effects of Exenatide on Obesity and Appetite in Overweight Patients With Prader-Willi Syndrome|
|Study Start Date :||March 2012|
|Actual Primary Completion Date :||May 2013|
|Actual Study Completion Date :||December 2013|
U.S. FDA Resources
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).
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).
Other Name: Byetta
- Change in Weight [ Time Frame: 6 months ]Change in weight (kg) after 6 months of treatment with study drug. Described as mean +/- SD
- % Change in Body Mass Index (BMI) [ Time Frame: 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.
- Change in BMI Z-Score [ Time Frame: 6 months ]
- Change in HbA1c (%) [ Time Frame: 6 months ]
- Change in Insulin Levels [ Time Frame: 6 months ]
- Change in Leptin [ Time Frame: 6 months ]
- Change in Acy Ghr [ Time Frame: 6 months ]
- Change in Pancreatic Peptide (PP) [ Time Frame: 6 months ]
- Appetite Scores [ Time Frame: 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
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01444898
|United States, California|
|Children's Hospital of Los Angeles|
|Los Angeles, California, United States, 90027|
|Principal Investigator:||Debra Jeandron, MD||Children's Hospital Los Angeles|