Liraglutide Use in Prader-Willi Syndrome

Study Overview
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Brief Summary
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Liraglutide is a glucagon like peptide -1 (GLP-1) agonist, which is approved for use in patients with type 2 diabetes. Studies of liraglutide have shown an appetite suppressive effect and has been associated with weight loss in patients with type 2 diabetes. Liraglutide use in the treatment of PWS is limited to one case report by Cyganek et el (See attached Citation). In this case report, the subject showed improvements in hemoglobin A1c and body weight over 14 weeks. The investigators plan to examine the use of liraglutide in a single subject with Prader Willi Syndrome and type 2 diabetes for one year. The investigators will examine clinical parameters, clinical assessment of hunger, and biochemical markers of diabetes and lipid control.

Conditions
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Diabetes Mellitus Type 2, Prader Willi Syndrome

Intervention / Treatment
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* Drug: Liraglutide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Prader Willi Syndrome, Diabetes Type 2 Exclusion Criteria: Previous or family history of Medullary Carcinoma of the Thyroid or multiple endocrine neoplasia syndrome. Subjects with acute or chronic Pancreatitis

Ages Eligible for Study
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Minimum Age: 19 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Liraglutide<br>Treatment of Diabetes Mellitus Type 2 with Liraglutide in the setting of Prader Willi Syndrome | Drug: Liraglutide<br>* Liraglutide 0.6 mg SQ daily for one week, increase to a maximum dose of 1.8 mg SQ daily by 6 weeks, and subsequent continuation for the remainder of a year.<br>* Other names: Victoza;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemoglobin A1C |  | Change from baseline in A1C at 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Fasting Blood Glucose (mmol/L) |  | Change from baseline in fasting blood glucose at 12 months | 
| Fasting Blood Insulin level |  | Change from baseline in fasting blood insulin at 12 months | 
| Body Weight (kg) |  | Change from baseline in body weight at 12 months | 
| Fasting Lipid Profile |  | Change from baseline in fasting lipid profile at 12 months | 
| Hip Circumference (cm) |  | Change from baseline in hip circumference at 12 months | 
| Total Body Adipose Tissue Distribution (whole body Computed Tomography) |  | Change from baseline in total body adipose tissue distribution at 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Diabetes Mellitus Type 2, Prader Willi Syndrome