Trial · Interventional

CompletedNot applicableResults postedRegistry data

Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome

NCTNCT01444898Other idCCI 11-00227

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Not applicable
Enrollment: 10 participants (actual)
Sponsor: Children's Hospital Los Angeles · Other
Design: Allocation not applicable · Open label (no masking)
Countries: 1 country
United States
Start: Mar 2012
Primary completion: May 2013
Completion: Dec 2013
First posted: 3 Oct 2011 (estimated)
Last update posted: 29 Sept 2016 (estimated)
Conditions: Prader-Willi Syndrome

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

201220132014

N/A
Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome
NCT01444898
Mar 2012 to Dec 2013

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

ExenatideDrug

Arms1

Experimental
Exenatide
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).

Primary outcomes9

Outcomes the sponsor listed as primary.

Change in Weight
Time frame · 6 months
Registry description
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
Registry description
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
Registry description
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

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· 6 months

Change in Weight

Group	Value (kg)	Spread
Exenatide	-.5	.9

Primary outcomeMean· 6 months

% Change in Body Mass Index (BMI)

Group	Value (% change in BMI)	Spread
Exenatide	1.3	2

Primary outcomeMean· 6 months

Change in BMI Z-Score

Group	Value (units on a scale)	Spread
Exenatide	.1	.4

Primary outcomeMean· 6 months

Change in HbA1c (%)

Group	Value (percentage)	Spread
Exenatide	-.3	.2

Primary outcomeMean· 6 months

Change in Insulin Levels

Group	Value (u/U ml^-1)	Spread
Exenatide	3	.5

Primary outcomeMean· 6 months

Change in Leptin

Group	Value (ng ml^-1)	Spread
Exenatide	-7.4	3.8

Primary outcomeMean· 6 months

Change in Acy Ghr

Group	Value (pg ml^-1)	Spread
Exenatide	263	99.5

Primary outcomeMean· 6 months

Change in Pancreatic Peptide (PP)

Group	Value (pg ml^-1)	Spread
Exenatide	15	6

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
ExenatideAll 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).	–	0 / 10	2 / 10

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies4

Studies that cite this trial.

Studies with results

3 studies on this page

Indexing hint: Clinical trial · 2017

6 results

Effects of exenatide on weight and appetite in overweight adolescents and young adults with Prader-Willi syndrome.

Salehi et al. 2017Pediatric obesitycited by 58

PMID 27071367PMC5288290DOI 10.1111/ijpo.12131PUB-27071367

Rights unknown6 results

Source link See results (6)

Indexing hint: Randomized controlled trial · 2010

3 results

Effects of exenatide and lifestyle modification on body weight and glucose tolerance in obese subjects with and without pre-diabetes.

Rosenstock et al. 2010Diabetes carecited by 172

PMID 20332357PMC2875418DOI 10.2337/dc09-1203PUB-20332357

Free to read · publisher termsCC BY-NC-ND3 results

Read at source See results (3)

PubMed tag: none · 2007

4 results

Exendin-4 potently decreases ghrelin levels in fasting rats.

Pérez-Tilve et al. 2007Diabetescited by 72

PMID 17192476DOI 10.2337/db05-0996PUB-17192476

Rights unknown4 results

Source link See results (4)

More studies

1 study on this page

Indexing hint: Review · 2006

The hypothalamus, hormones, and hunger: alterations in human obesity and illness.

Goldstone et al. 2006Progress in brain researchcited by 48

PMID 16876568DOI 10.1016/s0079-6123(06)53003-1PUB-16876568

Full text at the publisher

Publisher page

Eligibility

Who could join, as posted.

Sex: All
Age: from 13 Years up to 20 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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 \< 30 mL/min * Other syndromic diagnoses * gastrointestinal (GI) or renal illness in the 1 month prior to entering study * Inability to take study drug * Pregnancy * Initiation of growth hormone (GH), estrogen, or testosterone or change \> 25% of dose/kg/day during the 6 months prior to starting study * Non-English speaking

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