{"protocolSection":{"identificationModule":{"nctId":"NCT01444898","orgStudyIdInfo":{"id":"CCI 11-00227"},"organization":{"fullName":"Children's Hospital Los Angeles","class":"OTHER"},"briefTitle":"Effects of Exenatide on Overweight Adolescents With Prader-Willi Syndrome","officialTitle":"Effects of Exenatide on Obesity and Appetite in Overweight Patients With Prader-Willi Syndrome"},"statusModule":{"statusVerifiedDate":"2016-09","overallStatus":"COMPLETED","expandedAccessInfo":{"hasExpandedAccess":false},"startDateStruct":{"date":"2012-03"},"primaryCompletionDateStruct":{"date":"2013-05","type":"ACTUAL"},"completionDateStruct":{"date":"2013-12","type":"ACTUAL"},"studyFirstSubmitDate":"2011-09-27","studyFirstSubmitQcDate":"2011-09-30","studyFirstPostDateStruct":{"date":"2011-10-03","type":"ESTIMATED"},"resultsFirstSubmitDate":"2016-06-14","resultsFirstSubmitQcDate":"2016-09-27","resultsFirstPostDateStruct":{"date":"2016-09-29","type":"ESTIMATED"},"lastUpdateSubmitDate":"2016-09-27","lastUpdatePostDateStruct":{"date":"2016-09-29","type":"ESTIMATED"}},"sponsorCollaboratorsModule":{"responsibleParty":{"type":"PRINCIPAL_INVESTIGATOR","investigatorFullName":"Mitchell E. Geffner","investigatorTitle":"Principal Investigator","investigatorAffiliation":"Children's Hospital Los Angeles"},"leadSponsor":{"name":"Children's Hospital Los Angeles","class":"OTHER"}},"oversightModule":{"oversightHasDmc":false},"descriptionModule":{"briefSummary":"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.","detailedDescription":"BACKGROUND:\n\nPrader-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.\n\nOBJECTIVE: 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."},"conditionsModule":{"conditions":["Prader-Willi Syndrome"],"keywords":["Prader-Willi Syndrome","Obesity","Weight","Exenatide","Byetta","Pediatrics","Adolescents"]},"designModule":{"studyType":"INTERVENTIONAL","phases":["NA"],"designInfo":{"allocation":"NA","interventionModel":"SINGLE_GROUP","primaryPurpose":"TREATMENT","maskingInfo":{"masking":"NONE"}},"enrollmentInfo":{"count":10,"type":"ACTUAL"}},"armsInterventionsModule":{"armGroups":[{"label":"Exenatide","type":"EXPERIMENTAL","description":"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).","interventionNames":["Drug: Exenatide"]}],"interventions":[{"type":"DRUG","name":"Exenatide","description":"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).","armGroupLabels":["Exenatide"],"otherNames":["Byetta"]}]},"outcomesModule":{"primaryOutcomes":[{"measure":"Change in Weight","description":"Change in weight (kg) after 6 months of treatment with study drug. Described as mean +/- SD","timeFrame":"6 months"},{"measure":"% Change in Body Mass Index (BMI)","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.","timeFrame":"6 months"},{"measure":"Change in BMI Z-Score","timeFrame":"6 months"},{"measure":"Change in HbA1c (%)","timeFrame":"6 months"},{"measure":"Change in Insulin Levels","timeFrame":"6 months"},{"measure":"Change in Leptin","timeFrame":"6 months"},{"measure":"Change in Acy Ghr","timeFrame":"6 months"},{"measure":"Change in Pancreatic Peptide (PP)","timeFrame":"6 months"},{"measure":"Appetite Scores","description":"Appetite scores using a syndrome-validated hyperphagia questionnaire\n\n11 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.\n\nPossible ranges: Total 11-55, behavior 5-25, drive 4-20, severity 2-10","timeFrame":"6 months"}]},"eligibilityModule":{"eligibilityCriteria":"Inclusion Criteria:\n\n* Diagnosis of Prader Willi Syndrome confirmed by genetic testing (DNA methylation or FISH)\n* Ages 13-20 years\n* body mass index (BMI) \\> 85th percentile for age and gender\n\nExclusion Criteria:\n\n* Is currently using or has previously used a glucagon-like peptide-1 (GLP-1) agonist\n* History of pancreatitis, or renal failure\n* History of familial pancreatitis\n* Amylase, or lipase levels \\> 2.5 times the upper limit of normal any time in the previous 2 years\n* Creatinine clearance \\< 30 mL/min\n* Other syndromic diagnoses\n* gastrointestinal (GI) or renal illness in the 1 month prior to entering study\n* Inability to take study drug\n* Pregnancy\n* Initiation of growth hormone (GH), estrogen, or testosterone or change \\> 25% of dose/kg/day during the 6 months prior to starting study\n* Non-English speaking","healthyVolunteers":false,"sex":"ALL","minimumAge":"13 Years","maximumAge":"20 Years","stdAges":["CHILD","ADULT"]},"contactsLocationsModule":{"overallOfficials":[{"name":"Debra Jeandron, MD","affiliation":"Children's Hospital Los Angeles","role":"PRINCIPAL_INVESTIGATOR"}],"locations":[{"facility":"Children's Hospital of Los Angeles","city":"Los Angeles","state":"California","zip":"90027","country":"United States","geoPoint":{"lat":34.05223,"lon":-118.24368}}]},"referencesModule":{"references":[{"pmid":"16876568","type":"BACKGROUND","citation":"Goldstone AP. The hypothalamus, hormones, and hunger: alterations in human obesity and illness. Prog Brain Res. 2006;153:57-73. doi: 10.1016/S0079-6123(06)53003-1."},{"pmid":"14693421","type":"BACKGROUND","citation":"Goldstone AP. Prader-Willi syndrome: advances in genetics, pathophysiology and treatment. Trends Endocrinol Metab. 2004 Jan-Feb;15(1):12-20. doi: 10.1016/j.tem.2003.11.003."},{"pmid":"20424341","type":"BACKGROUND","citation":"Suzuki K, Simpson KA, Minnion JS, Shillito JC, Bloom SR. The role of gut hormones and the hypothalamus in appetite regulation. Endocr J. 2010;57(5):359-72. doi: 10.1507/endocrj.k10e-077. Epub 2010 Apr 14."},{"pmid":"11739476","type":"BACKGROUND","citation":"Wren AM, Seal LJ, Cohen MA, Brynes AE, Frost GS, Murphy KG, Dhillo WS, Ghatei MA, Bloom SR. Ghrelin enhances appetite and increases food intake in humans. J Clin Endocrinol Metab. 2001 Dec;86(12):5992. doi: 10.1210/jcem.86.12.8111."},{"pmid":"12091883","type":"BACKGROUND","citation":"Cummings DE, Clement K, Purnell JQ, Vaisse C, Foster KE, Frayo RS, Schwartz MW, Basdevant A, Weigle DS. Elevated plasma ghrelin levels in Prader Willi syndrome. Nat Med. 2002 Jul;8(7):643-4. doi: 10.1038/nm0702-643. No abstract available."},{"pmid":"15292322","type":"BACKGROUND","citation":"Paik KH, Jin DK, Song SY, Lee JE, Ko SH, Song SM, Kim JS, Oh YJ, Kim SW, Lee SH, Kim SH, Kwon EK, Choe YH. Correlation between fasting plasma ghrelin levels and age, body mass index (BMI), BMI percentiles, and 24-hour plasma ghrelin profiles in Prader-Willi syndrome. J Clin Endocrinol Metab. 2004 Aug;89(8):3885-9. doi: 10.1210/jc.2003-032137."},{"pmid":"20332357","type":"BACKGROUND","citation":"Rosenstock J, Klaff LJ, Schwartz S, Northrup J, Holcombe JH, Wilhelm K, Trautmann M. Effects of exenatide and lifestyle modification on body weight and glucose tolerance in obese subjects with and without pre-diabetes. Diabetes Care. 2010 Jun;33(6):1173-5. doi: 10.2337/dc09-1203. Epub 2010 Mar 23."},{"pmid":"17192476","type":"BACKGROUND","citation":"Perez-Tilve D, Gonzalez-Matias L, Alvarez-Crespo M, Leiras R, Tovar S, Dieguez C, Mallo F. Exendin-4 potently decreases ghrelin levels in fasting rats. Diabetes. 2007 Jan;56(1):143-51. doi: 10.2337/db05-0996."},{"pmid":"21227526","type":"BACKGROUND","citation":"Seetho IW, Jones G, Thomson GA, Fernando DJ. Treating diabetes mellitus in Prader-Willi syndrome with Exenatide. Diabetes Res Clin Pract. 2011 Apr;92(1):e1-2. doi: 10.1016/j.diabres.2010.12.009. Epub 2011 Jan 11."},{"pmid":"27071367","type":"RESULT","citation":"Salehi P, Hsu I, Azen CG, Mittelman SD, Geffner ME, Jeandron D. Effects of exenatide on weight and appetite in overweight adolescents and young adults with Prader-Willi syndrome. Pediatr Obes. 2017 Jun;12(3):221-228. doi: 10.1111/ijpo.12131. Epub 2016 Apr 13."}]},"ipdSharingStatementModule":{"ipdSharing":"UNDECIDED"}},"resultsSection":{"participantFlowModule":{"groups":[{"id":"FG000","title":"Exenatide","description":"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)."}],"periods":[{"title":"Overall Study","milestones":[{"type":"STARTED","achievements":[{"groupId":"FG000","numSubjects":"10"}]},{"type":"COMPLETED","achievements":[{"groupId":"FG000","numSubjects":"9"}]},{"type":"NOT COMPLETED","achievements":[{"groupId":"FG000","numSubjects":"1"}]}],"dropWithdraws":[{"type":"Withdrawal by Subject","reasons":[{"groupId":"FG000","numSubjects":"1"}]}]}]},"baselineCharacteristicsModule":{"groups":[{"id":"BG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"BG000","value":"10"}]}],"measures":[{"title":"Age, Categorical","paramType":"COUNT_OF_PARTICIPANTS","unitOfMeasure":"Participants","classes":[{"categories":[{"title":"<=18 years","measurements":[{"groupId":"BG000","value":"6"}]},{"title":"Between 18 and 65 years","measurements":[{"groupId":"BG000","value":"4"}]},{"title":">=65 years","measurements":[{"groupId":"BG000","value":"0"}]}]}]},{"title":"Age, Continuous","paramType":"MEDIAN","dispersionType":"FULL_RANGE","unitOfMeasure":"years","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"16","lowerLimit":"14.7","upperLimit":"24.6"}]}]}]},{"title":"Sex: Female, Male","paramType":"COUNT_OF_PARTICIPANTS","unitOfMeasure":"Participants","classes":[{"categories":[{"title":"Female","measurements":[{"groupId":"BG000","value":"6"}]},{"title":"Male","measurements":[{"groupId":"BG000","value":"4"}]}]}]},{"title":"Ethnicity (NIH/OMB)","paramType":"COUNT_OF_PARTICIPANTS","unitOfMeasure":"Participants","classes":[{"categories":[{"title":"Hispanic or Latino","measurements":[{"groupId":"BG000","value":"1"}]},{"title":"Not Hispanic or Latino","measurements":[{"groupId":"BG000","value":"6"}]},{"title":"Unknown or Not Reported","measurements":[{"groupId":"BG000","value":"3"}]}]}]},{"title":"Race (NIH/OMB)","paramType":"COUNT_OF_PARTICIPANTS","unitOfMeasure":"Participants","classes":[{"categories":[{"title":"American Indian or Alaska Native","measurements":[{"groupId":"BG000","value":"0"}]},{"title":"Asian","measurements":[{"groupId":"BG000","value":"0"}]},{"title":"Native Hawaiian or Other Pacific Islander","measurements":[{"groupId":"BG000","value":"0"}]},{"title":"Black or African American","measurements":[{"groupId":"BG000","value":"1"}]},{"title":"White","measurements":[{"groupId":"BG000","value":"6"}]},{"title":"More than one race","measurements":[{"groupId":"BG000","value":"2"}]},{"title":"Unknown or Not Reported","measurements":[{"groupId":"BG000","value":"1"}]}]}]},{"title":"Region of Enrollment","description":"Region of Enrollment = California","paramType":"NUMBER","unitOfMeasure":"participants","classes":[{"title":"United States","categories":[{"measurements":[{"groupId":"BG000","value":"10"}]}]}]},{"title":"Weight","description":"Weight (kg)","paramType":"MEAN","dispersionType":"STANDARD_DEVIATION","unitOfMeasure":"kg","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"102.5","spread":"18.6"}]}]}]},{"title":"BMI","description":"BMI (kg m\\^-2)","paramType":"MEDIAN","dispersionType":"INTER_QUARTILE_RANGE","unitOfMeasure":"kg m^-2","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"41.7","lowerLimit":"34.1","upperLimit":"55"}]}]}]},{"title":"BMI Z-Score","paramType":"MEAN","dispersionType":"STANDARD_DEVIATION","unitOfMeasure":"units on a scale","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"3.5","spread":"1.1"}]}]}]},{"title":"Glycosylated hemoglobin (HbA1c)","description":"Glycosylated hemoglobin (HbA1c), percentage of total hemoglobin","paramType":"MEDIAN","dispersionType":"INTER_QUARTILE_RANGE","unitOfMeasure":"percentage of total hemoglobin","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"5.9","lowerLimit":"5.6","upperLimit":"7.9"}]}]}]},{"title":"Insulin","description":"Insulin (u/U ml\\^-1)","paramType":"MEDIAN","dispersionType":"INTER_QUARTILE_RANGE","unitOfMeasure":"uIU ml^-1","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"10.5","lowerLimit":"6","upperLimit":"21"}]}]}]},{"title":"Leptin","description":"Leptin (ng ml \\^-1)","paramType":"MEAN","dispersionType":"STANDARD_DEVIATION","unitOfMeasure":"ng ml ^-1","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"36.4","spread":"18.3"}]}]}]},{"title":"Acy Ghr","description":"Acylated ghrelin, Acy Ghr (pg ml \\^-1)","paramType":"MEDIAN","dispersionType":"INTER_QUARTILE_RANGE","unitOfMeasure":"pg ml ^-1","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"362.3","lowerLimit":"259","upperLimit":"740.7"}]}]}]},{"title":"PP","description":"Pancreatic polypeptide, PP (pg ml \\^-1)","paramType":"MEDIAN","dispersionType":"INTER_QUARTILE_RANGE","unitOfMeasure":"pg ml ^-1","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"89","lowerLimit":"31","upperLimit":"662"}]}]}]},{"title":"Appetite","description":"Appetite scores - For 1 and 2: 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.\n\nPossible ranges: Total 11-55, behavior 5-25, drive 4-20, severity 2-10","paramType":"MEAN","dispersionType":"STANDARD_DEVIATION","unitOfMeasure":"units on a scale","classes":[{"title":"Total Appetite Score","categories":[{"measurements":[{"groupId":"BG000","value":"32.2","spread":"8.7"}]}]},{"title":"Appetite Behavior","categories":[{"measurements":[{"groupId":"BG000","value":"14.5","spread":"4.9"}]}]},{"title":"Appetite Drive","categories":[{"measurements":[{"groupId":"BG000","value":"12.6","spread":"3.1"}]}]},{"title":"Appetite Severity","categories":[{"measurements":[{"groupId":"BG000","value":"5.1","spread":"1.5"}]}]}]},{"title":"Adiposity","description":"Truncal fat as measured by dual x-ray absorptiometry","paramType":"NUMBER","unitOfMeasure":"percentage of fat","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"44.7"}]}]}]},{"title":"Adiposity","description":"Mean truncal fat","paramType":"MEAN","dispersionType":"STANDARD_DEVIATION","unitOfMeasure":"kg","classes":[{"categories":[{"measurements":[{"groupId":"BG000","value":"20","spread":"4.6"}]}]}]}]},"outcomeMeasuresModule":{"outcomeMeasures":[{"type":"PRIMARY","title":"Change in Weight","description":"Change in weight (kg) after 6 months of treatment with study drug. Described as mean +/- SD","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"kg","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"-.5","spread":".9"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".07","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"% Change in Body Mass Index (BMI)","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.","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"% change in BMI","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"1.3","spread":"2"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":"0.08","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Change in BMI Z-Score","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"units on a scale","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":".1","spread":".4"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".8","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Change in HbA1c (%)","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"percentage","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"-.3","spread":".2"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".04","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Change in Insulin Levels","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"u/U ml^-1","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"3","spread":".5"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".8","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Change in Leptin","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"ng ml^-1","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"-7.4","spread":"3.8"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".2","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Change in Acy Ghr","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"pg ml^-1","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"263","spread":"99.5"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".2","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Change in Pancreatic Peptide (PP)","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"pg ml^-1","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"categories":[{"measurements":[{"groupId":"OG000","value":"15","spread":"6"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".04","statisticalMethod":"Mixed Models Analysis"}]},{"type":"PRIMARY","title":"Appetite Scores","description":"Appetite scores using a syndrome-validated hyperphagia questionnaire\n\n11 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.\n\nPossible ranges: Total 11-55, behavior 5-25, drive 4-20, severity 2-10","reportingStatus":"POSTED","paramType":"MEAN","dispersionType":"Standard Deviation","unitOfMeasure":"units on a scale","timeFrame":"6 months","groups":[{"id":"OG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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)."}],"denoms":[{"units":"Participants","counts":[{"groupId":"OG000","value":"10"}]}],"classes":[{"title":"Total Appetite Score Baseline","categories":[{"measurements":[{"groupId":"OG000","value":"32.2","spread":"8.7"}]}]},{"title":"Total Appetite Score 6 month","categories":[{"measurements":[{"groupId":"OG000","value":"25.4","spread":"7.2"}]}]},{"title":"Behavior Score Baseline","categories":[{"measurements":[{"groupId":"OG000","value":"14.5","spread":"4.9"}]}]},{"title":"Behavior Score 6 months","categories":[{"measurements":[{"groupId":"OG000","value":"10.6","spread":"3.8"}]}]},{"title":"Drive Score Baseline","categories":[{"measurements":[{"groupId":"OG000","value":"12.6","spread":"3.1"}]}]},{"title":"Drive Score 6 months","categories":[{"measurements":[{"groupId":"OG000","value":"10.4","spread":"2.7"}]}]},{"title":"Severity Score Baseline","categories":[{"measurements":[{"groupId":"OG000","value":"5.1","spread":"1.5"}]}]},{"title":"Severity Score 6 months","categories":[{"measurements":[{"groupId":"OG000","value":"4.4","spread":"2.2"}]}]}],"analyses":[{"groupIds":["OG000"],"testedNonInferiority":false,"nonInferiorityType":"SUPERIORITY_OR_OTHER","pValue":".004","statisticalMethod":"Mixed Models Analysis"}]}]},"adverseEventsModule":{"frequencyThreshold":"5","eventGroups":[{"id":"EG000","title":"Exenatide","description":"All subjects enrolled in this study will be given Exenatide for 6 months.\n\nExenatide: 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).","seriousNumAffected":0,"seriousNumAtRisk":10,"otherNumAffected":2,"otherNumAtRisk":10}],"otherEvents":[{"term":"abdominal discomfort with diarrhea","organSystem":"Gastrointestinal disorders","notes":"One subject reported abdominal cramping and diarrhea for a short period because of acute infectious gastroenteritis. Another subject experienced mild abdominal discomfort and diarrhea after doubling the dose of exenatide for 1 d.","stats":[{"groupId":"EG000","numEvents":2,"numAffected":2,"numAtRisk":10}]}]},"moreInfoModule":{"limitationsAndCaveats":{"description":"* small sample size\n* referral bias due to small recruitment area\n* confounding factors during the study, including variations in eating schedule, psychotropic medications, caretaker supervision and activity"},"certainAgreement":{"piSponsorEmployee":false,"restrictiveAgreement":false},"pointOfContact":{"title":"Dr. Mitchell Geffner","organization":"Children's Hospital Los Angeles","email":"mgeffner@chla.usc.edu","phone":"323-361-7032"}}},"derivedSection":{"miscInfoModule":{"versionHolder":"2026-10-09"},"conditionBrowseModule":{"meshes":[{"id":"D011218","term":"Prader-Willi Syndrome"},{"id":"D009765","term":"Obesity"},{"id":"D001835","term":"Body Weight"}],"ancestors":[{"id":"D008607","term":"Intellectual Disability"},{"id":"D019954","term":"Neurobehavioral Manifestations"},{"id":"D009461","term":"Neurologic Manifestations"},{"id":"D009422","term":"Nervous System Diseases"},{"id":"D000015","term":"Abnormalities, Multiple"},{"id":"D000013","term":"Congenital Abnormalities"},{"id":"D009358","term":"Congenital, Hereditary, and Neonatal Diseases and Abnormalities"},{"id":"D025063","term":"Chromosome Disorders"},{"id":"D030342","term":"Genetic Diseases, Inborn"},{"id":"D000096803","term":"Imprinting Disorders"},{"id":"D050177","term":"Overweight"},{"id":"D044343","term":"Overnutrition"},{"id":"D009748","term":"Nutrition Disorders"},{"id":"D009750","term":"Nutritional and Metabolic Diseases"},{"id":"D012816","term":"Signs and Symptoms"},{"id":"D013568","term":"Pathological Conditions, Signs and Symptoms"}]},"interventionBrowseModule":{"meshes":[{"id":"D000077270","term":"Exenatide"}],"ancestors":[{"id":"D010455","term":"Peptides"},{"id":"D000602","term":"Amino Acids, Peptides, and Proteins"},{"id":"D014688","term":"Venoms"},{"id":"D045424","term":"Complex Mixtures"},{"id":"D014118","term":"Toxins, Biological"},{"id":"D001685","term":"Biological Factors"}]}},"hasResults":true}