Clinical trial NCT01542242: Liraglutide Use in Prader-Willi Syndrome
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Diabetes Mellitus Type 2
Prader Willi Syndrome
Keywords: Diabetes Mellitus Type 2
Prader Willi Syndrome
Lead sponsor: Vancouver General Hospital
Sponsor class: OTHER
Collaborators: name: Novo Nordisk A/S
First posted: 2012-03-02
Enrollment: 1
Sex: MALE
Minimum age (years): 19
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Hemoglobin A1C
timeFrame: Change from baseline in A1C at 12 months
Secondary outcomes:
measure: Fasting Blood Glucose (mmol/L)
timeFrame: Change from baseline in fasting blood glucose at 12 months
measure: Fasting Blood Insulin level
timeFrame: Change from baseline in fasting blood insulin at 12 months
measure: Body Weight (kg)
timeFrame: Change from baseline in body weight at 12 months
measure: Fasting Lipid Profile
timeFrame: Change from baseline in fasting lipid profile at 12 months
measure: Hip Circumference (cm)
timeFrame: Change from baseline in hip circumference at 12 months
measure: Total Body Adipose Tissue Distribution (whole body Computed Tomography)
timeFrame: Change from baseline in total body adipose tissue distribution at 12 months
Other outcomes:
Locations:
city: Vancouver
country: Canada
facility: Vancouver General Hospital - Diamond Center
state: British Columbia
Study officials:
affiliation: Vancouver General Hospital, University of British Columbia
name: Jason Kong, MD, FRCPC
role: PRINCIPAL_INVESTIGATOR
References:
citation: Cyganek K, Koblik T, Kozek E, Wojcik M, Starzyk J, Malecki MT. Liraglutide therapy in Prader-Willi syndrome. Diabet Med. 2011 Jun;28(6):755-6. doi: 10.1111/j.1464-5491.2011.03280.x. No abstract available.
citation: Sze L, Purtell L, Jenkins A, Loughnan G, Smith E, Herzog H, Sainsbury A, Steinbeck K, Campbell LV, Viardot A. Effects of a single dose of exenatide on appetite, gut hormones, and glucose homeostasis in adults with Prader-Willi syndrome. J Clin Endocrinol Metab. 2011 Aug;96(8):E1314-9. doi: 10.1210/jc.2011-0038. Epub 2011 Jun 1.