N/A
N=10
Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome
Prader-Willi Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01444898 ↗Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Sep 2016
Primary outcome: Primary: Change in Weight — -.5 kg — p=.07
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- Exenatide (Drug)
- Age
- Pediatric, Adult · 13+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Los Angeles
- Primary completion
- May 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Weight |
-.5 | .07 |
| PRIMARY % Change in Body Mass Index (BMI) |
1.3 | 0.08 |
| PRIMARY Change in BMI Z-Score |
.1 | .8 |
| PRIMARY Change in HbA1c (%) |
-.3 | .04 sig |
| PRIMARY Change in Insulin Levels |
3 | .8 |
| PRIMARY Change in Leptin |
-7.4 | .2 |
| PRIMARY Change in Acy Ghr |
263 | .2 |
| PRIMARY Change in Pancreatic Peptide (PP) |
15 | .04 sig |
| PRIMARY Appetite Scores |
32.2; 25.4; 14.5; 10.6; 12.6; 10.4 | .004 sig |
Summary
Prader-Willi Syndrome (PWS) is one of the most common genetic causes of obesity. Obesity is a major source of morbidity and mortality in this population. It can lead to sleep apnea, cor pulmonale, diabetes mellitus, and atherosclerosis. PWS has distinct characteristics that set it apart from other forms of obesity including insatiable appetite and food-seeking behavior which can be disruptive to home and school activities, and can cause severe social and psychological turmoil within families. PWS is also associated with unique hormonal abnormalities, most notably hyperghrelinemia. Ghrelin is a gut hormone produced in the stomach that stimulates food intake during a fast. It is hypothesized that the extremely high ghrelin levels in patients with PWS may cause or contribute to their insatiable appetite. Exenatide, a medication used in the treatment of type 2 diabetes mellitus in adults, appears to suppress ghrelin levels and cause weight loss. It was designed to mimic glucagon-like peptide 1 (GLP-1), an incretin hormone that stimulates insulin secretion and delays gastric emptying, among other effects. In the present study, the investigators will investigate the effects of a 6 month trial of exenatide in overweight adolescents with PWS. The investigators will quantify the changes in weight and body composition, as well as subjective measures of appetite, and concentrations of appetite-associated hormones. The investigators hypothesize that exenatide will improve weight, body composition, appetite, and plasma ghrelin levels during the treatment period.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Prader Willi Syndrome confirmed by genetic testing (DNA methylation or FISH)
- Ages 13-20 years
- body mass index (BMI) > 85th percentile for age and gender
Exclusion Criteria
- Is currently using or has previously used a glucagon-like peptide-1 (GLP-1) agonist
- History of pancreatitis, or renal failure
- History of familial pancreatitis
- Amylase, or lipase levels > 2.5 times the upper limit of normal any time in the previous 2 years
- Creatinine clearance 25% of dose/kg/day during the 6 months prior to starting study
- Non-English speaking
Data sourced from ClinicalTrials.gov (NCT01444898). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.