Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|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|
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|