Clinical Trial: Prader-Willi Syndrome and Appetite
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: Effect of Somatostatin on Ghrelin Concentrations, Food Seeking Behaviour and Weight in Patients With Prader-Willi Syndrome
Brief Summary: Excessive weight gain is a cardinal feature of Prader-Willi syndrome (PWS) for which there is presently no effective treatment. It is caused by increased appetite, decreased perception of satiety and obsessive and compulsive behaviour towards food. Ghrelin is a powerful appetite-stimulating hormone. Patients with PWS have markedly elevated ghrelin levels, suggesting that it may be responsible for the increased food intake. The goal of the study is to determine whether treatment with somatostatin (Sandostatin), a hormone that inhibits ghrelin, is an effective treatment for the prevention and treatment of weight excess in patients with PWS.
Detailed Summary:
Prader-Willi Syndrome and Obesity:
Prader-Willi syndrome (PWS) is a genetic disorder occurring in 1/10,000 to 1/15,000 live births. Clinical characteristics include neonatal and infantile central hypotonia with feeding problems and poor weight gain followed after 1-3 years by hyperphagia and excessive weight gain. Patients also have characteristic facial features, short stature (possibly due to growth hormone [GH] deficiency of hypothalamic origin), hypogonadism, increased pain threshold, global developmental delay and variable mental retardation.
Obesity is clearly one of the cardinal features of PWS. It can result in tremendous distress for both the parents and the affected child and is a significant health problem with complications that include hypertension, sleep apneas and diabetes. More than 1/3 of patients with PWS weigh more than 200% of their ideal body weight. Obesity is thought to result mainly from hyperphagia, persistent hunger with increased caloric intake, decreased perception of satiety and obsessive and compulsive behaviours that are primarily food related. Decreased physical activity associated with hypotonia and/or decreased energy expenditure is also thought to play a role in the pathophysiology of obesity.
The treatment of obesity in PWS is very difficult and requires constant involvement of the parents or caregivers with establishment of rigid structures around the young patient: increased physical activity, hiding food, locking fridges etc. Recently, studies on the effects of growth hormone (GH) treatment (now an approved indication in most patients with PWS in the US irrespective of their GH status) on body composition in PWS have been performed. While GH, among other favourable effects, significantly improves body composition (increased lean mass a
Sponsor: University of British Columbia
Current Primary Outcome: Changes in ghrelin concentrations during a test meal [ Time Frame: 8 to 10 AM ]
Original Primary Outcome: Changes in ghrelin concentrations during a mealtest (8 to 10 AM)
Current Secondary Outcome: Change in weight, behaviour and food intake
Original Secondary Outcome: Same as current
Information By: University of British Columbia
Dates:
Date Received: September 9, 2005
Date Started: August 2004
Date Completion:
Last Updated: October 29, 2007
Last Verified: October 2007
