Saturday, November 19, 2011

Research Opportunity!

With a rare disease like PWS, there is a limited population for research. There is not much that Dean can qualify for at his age, but we look forward to being part of various trials as he grows older. If that makes it sound like we're eager to be guinea pigs, not really. It's just that again, with a limited population of people to draw from, these studies need whoever they can get. In the long run, it benefits all of us. Here's an opportunity!

Does exenatide help lead to weight loss in overweight, adolescents with Prader-Willi Syndrome?

Exenatide (Byetta; Amylin Pharmaceuticals, San Diego, CA) is a subcutaneous injection given twice a day which has been shown to lead to weight loss in adults. We are starting a study to see the effects of exenatide on obesity factors in overweight patients with Prader-Willi Syndrome between the ages of 13-20 over a 6 month period. The study will involve 5 visits to Children’s Hospital of Los Angeles (CHLA). There will be an initial screening visit which involves blood tests and if the patient qualifies, 4 more visits will be scheduled at CHLA. All visits will include physical exams, blood draws, as well as an appetite questionnaire. In addition, two of the visits will include a mixed meal tolerance test (MMTT) and a DEXA scan. The MMTT involves placing an IV and a series of 4 blood tests over 2 hours. The DEXA scan is an x-ray that will measure body fat.

The study is being conducted by Dr. Debra Jeandron and Dr. Parisa Salehi from the CHLA Endocrinology department. The following describes the requirements of the study. If you are interested, please contact us to discuss this study further.

To participate in this study, you:
Must have Prader-Willi Syndrome
Must be 13-20 years old
Must have a body mass index > 85th percentile
Must NOT be using or have used exenatide or a related medication
Must NOT have a current of past history of pancreas or kidney illness or problems
Must NOT have another diagnoses which may affect taking the medication or its results

$50 will be provided at each visit as compensation for you participation

Contact Person:
Parisa Salehi, MD
Office: (323) 361-8816

Study Location:
Children’s Hospital Los Angeles
4650 Sunset Blvd
Los Angeles, CA 90027

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