Participants Needed for PWS Studies
The National Institutes of Health maintains a searchable database that provides patients, family members and the public with information about current clinical research studies, see: clinicaltrials.gov. Click here to search this database for current and recently completed clinical trials relevant to people with Prader-Willi syndrome
Angelman, Rett, and Prader-Willi Syndromes Consortium, sponsored by the NIH Office of Rare Diseases Research, maintains a contact registry for individuals with these disorders. Please consider joining the registry to be notified of applicable clinical trials. An ongoing study Characteristics of Prader-Willi Syndrome and Early-Onset Morbid Obesity is recruiting participants.
Additional clinical trials that may be of interest to those with PWS:
Please contact each investigator or study coordinator directly for complete details, which will help you evaluate whether the trial is right for you.
Extraction of Neuronal Stem Cells from Dental Pulp for Human Neurogenetic Disease Studies
On-Line Temper Outburst Survey - University of Sydney
Dr. Steward Einfeld of the Brain & Mind Institute, University of Sydney, is studying temper outburst behavior in Prader-Willi syndrome, and has developed an online survey to better understand this behavior. To learn more and participate, please click here.
Participants Needed for PWS Research, Alberta Canada
Dr. Andrea Haqq (a Pediatric Endocrinologist at the University of Alberta) is recruiting children with PWS 5‐17 years of age for three research studies. Children 5‐17 years of age without PWS are also being recruited for all studies.
Study One: The purpose of this study is to determine if there is a specific type of meal (such as high protein‐low fat or high protein low carbohydrate) that would promote satiety in children with PWS. This study involves coming to the University of Alberta (in Edmonton) for three visits (each visit separated by 2 weeks).
Study Two: Dr. Haqq’s recent research has shown that children with PWS have lower levels of a peptide that is involved in neurological development (called brain‐derived neurotrophic factor or BDNF). The purpose of this study is to determine if the level of BDNF in blood is associated with eating behaviors and cognitive function in children with PWS. This study involves coming to the University of Alberta for one day. Study Two is funded by FPWR.
Study Three: The purpose of this study is to examine if autonomic nervous system function, the distribution of body fat, and level of hormones in blood is different in children with PWS. This study involves coming to the University of Alberta for two days.
For more information about these studies contact Dr. Haqq’s research coordinator Michelle Mackenzie at 780‐407‐7241 or email@example.com
New study of Behavior in Prader-Willi Syndrome- (7/01/2010)
This new NICHD-funded study is a continuation of the current Elisabeth Dykens PWS study to characterize and accurately describe the behavioral features of Prader-Willi Syndrome (PWS) and how these can be different based on age, gender, family history and genetic subtype of PWS. The study looks at key psychiatric features of PWS, like rigid and repetitive behaviors, insistence on sameness, tantrums, aggression and depression and how these change over a lifespan. We want to see if intervention timing is related to successful outcomes and effectiveness. We hope to see 170 families with children, teens and adults with PWS for a one day visit at Vanderbilt Kennedy Center in Nashville TN over the next 5 years. We want to see how children with PWS change and we will follow them every other year to track changes. Parents will receive a written feedback report of all results and behavioral management tips during visit. Parents who are interested should contact Elizabeth Roof at firstname.lastname@example.org or 615-343-3330 to get more information about the study.
The Effect of Growth Hormone Replacement Therapy on Physical and Behavioral Sexual Development in Persons with Prader-Willi Syndrome
Drs. Myers and Whitman of St. Louis University are performing a study to examine the impact of growth hormone replacement therapy (GHRT) on the sexual/gonadal maturation and functioning, and sexual behavior of males and females with Prader-Willi syndrome (PWS). This study is supported by FPWR. The subject population will include 20 adults (10 males and 10 females ages 18 years and over) and 20 youngsters (10 males and 10 females between the ages of 10-14 years) with Prader-Willi syndrome. Adults will be divided between 10 adults previously treated with growth hormone replacement therapy (GHRT) and 10 naive to GHRT. Youngsters will be drawn from those currently receiving GHRT. GH is provided, free of charge, by Pfizer.
A small stipend towards travel may be available for some families. Also Dr. Whitman has recently received another grant from PWSA for studying the hyperphagia and your child can also help out by participating in this new study at the same time if s/he is interested.
For more information about this study, please contact:
Dr. Barbara Whitman
Saint Louis University School of Medicine
Exercise in Children with Prader- Willi Syndrome
Researchers in the Kinesiology Department at California State University Fullerton (CSUF), in collaboration with the Children’s Hospital of Orange County (CHOC) located in the city of Orange in CA, are investigating the hormonal and metabolic responses to exercise in children with PWS and without PWS. The purpose of this study is to characterize these responses to exercise based on body fat or the genetics of PWS.
We intend to recruit youth with PWS between the ages 8-18 years old for a three-visit study (1.5 hours for the 1st visit to CHOC, 1 hour for a visit to CSUF, and 3 hours for a 2nd visit to CHOC). During both visits to CHOC the children will complete resistance exercises while wearing a weighted vest, in the 1st visit they will complete a health screening, and in the 2nd visit they will have their blood drawn. The visit to CSUF is for a body composition x-ray scan. Participating youth will be compensated with play equipment (worth $10) and a gift card worth $25 for their time, and parents/caregivers for mileage and parking.
For more information or to participate in this study, please contact Dr. Daniela Rubin by phone (714-278-4704) or by email at email@example.com or Dr. Dan Judelson at 714-278-5423 or by email at firstname.lastname@example.org. Hablamos español.
Prader- Willi Syndrome and Early Onset Morbid Obesity Natural History - Children's Hospital of Orange County
The purpose of the study is to collect natural history information on Prader-Willi syndrome and early onset morbid obesity to learn more about how these conditions can affect a person throughout his or her life from birth to an adult. This will mean better management and treatment in the future. The study takes about 4 hours and will include an interview, physical exam, blood draw, bone scan, intelligence and behavioral tests. This study is one of the sites for the Prader Willi Study that is part of the Angelman, Rett and Prader Willi Syndromes Consortium.
Children’s Hospital of Orange County
Contact: Virginia Kimonis, MD, Professor of Pediatrics,
University of California, Irvine (949) 824 0521/0571;