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Research for Prader-Willi syndrome

For many of them, they are dependent on your help. Please consider participating in one or more of their research projects. 

Help us to help you!
We need your participation

Study Name: 5202 - Prader-Willi Syndrome and Early-onset Morbid Obesity Natural History Clinical Protocol

Study Status: Recruiting

For:  Prader-Willi Syndrome

Summary:

Please Note: The Rare Diseases Clinical Research Network will make every effort to enroll all the patients we can, but we cannot make any guarantees that we will be able to enroll everyone in a particular study who wants to participate.

The purposes of this study are to

  • evaluate effects of growth hormone treatment in people with Prader-Willi Syndrome (PWS) when started at difference ages,

  • compare people with PWS to people with early-onset morbid obesity (EMO), and

  • collect information to use as a foundation for future research studies. Information gained from this study may help tailor long term management for people with PWS and EMO.

Target Enrollment:

The PWS group must be under 60 years of age and have had appropriate testing to confirm a diagnosis of PWS. The EMO group must be under 30 years of age and have a) documented medical history of weight exceeding 150% of Ideal Body Weight (IBW) or b) a Body Mass Index (BMI) of greater than 97% before four (4) years of age. The study will last five (5) years. You must be able to travel (at your own expense) to a Clinical Center once a year for evaluation.  

How to Participate:

In order to participate in a study, you must personally contact the study coordinator of any of the participating institutions by phone or by e-mail. Please use the information below to inquire about participation.

Kansas University Medical Center (ARPWS)
Kansas City, Kansas
Principal Investigator:  Merlin Butler, MD
Contact Person:  Marilyn Logan, RN
Office: 913-588-1315
Email: mlogan@kumc.edu

Help us to help you!  We need your participation in the Rare Disease Research Network consortium studying Prader-Willi syndrome.

The purpose of our study is to identify the natural history of PWS and those with EMO or early-onset of childhood morbid obesity and to learn more about the genetic causes and co-morbidities of Prader-Willi syndrome and to compare PWS with other causes of EMO. Eligible participants must have a confirmed genetic diagnosis of Prader-Willi syndrome or those with EMO have onset of obesity before 4 years of age. Individuals of all ages are eligible. For more information, please contact, Merlin G. Butler, MD, PhD: 913-588-1873, email: mbutler4@kumc.edu, Erin L. Youngs, MS: 913-588-1871, email: eyoungs@kumc.edu  or Marilyn Logan, RN: 913-588-1315, email: mlogan@kumc.edu 

University of California at Irvine (ARPWS)
Orange, California
Principal Investigator:  Virginia Kimonis, MD
Contact Person:  Virginia Kimonis, MD
Office:  714-456-2942
Email:  vkimonis@uci.edu

University of Florida Health Sciences Center (ARPWS)
Gainesville, Florida
Principal Investigator:  Daniel J. Driscoll, MD, PhD
Contact Person:  Christy Hollywood Lynn, RD, LDN
Office: 352-392-9789
Email: christyhollywood@peds.ufl.edu

Vanderbilt University Medical Center (ARPWS)
Nashville, Tennessee
Principal Investigator:  Elisabeth Dykens, Ph.D
Contact Person:  Elizabeth Roof, M.A., L.P.E.
Office: 615-343-3330
Email: elizabeth.roof@vanderbilt.edu

 

The Rare Diseases Clinical Research Network (RDCRN) was established by the National Institutes of Health (NIH) to develop research studies for rare diseases, and to encourage cooperative partnerships among researchers at the Network's clinical centers around the world. This increased cooperation may lead to discoveries that will help treat and perhaps prevent these rare diseases, as well as produce medical advances that will benefit the population in general.

 

The Rare Diseases Clinical Research Network is comprised of a Data Management Coordinating Center and 19 consortia (groups) studying over 90 rare diseases and Prader-Willi syndrome is one of them.

What is a registry?

The Rare Disease Network - A registry is a database of information about patients. Currently, the RDCRN hosts a contact registry of self reported, mostly demographic information about patients. The main goal of the registry is to disseminate information to patients and their families and communicate information about current and upcoming clinical trials.

PWSA (USA) Ė This registry is a database of information about people with Prader-Willi syndrome (PWS). The information can be updated by using a code identification (assigned after initial entry) and the personís date of birth. The information provides statistical information to researchers about the concerns of our individuals. At no time is detailed contact information released without your permission. When research is being conducted, PWSA (USA) may contact entries about the research giving you the opportunity to contact the researcher if you so desire.

Do I have to join the RDCRN registry?

The RDCRN offers the registry to all consortia in the Network. Patients and their families may access the Registry from the RDCRN web site. Each consortium may determine the degree to which they would like to use the registry to communicate to registrants about disease information, events or trials.

How does PWSA (USA)ís registry interact with the RDCRN registry?

For patient privacy protection, at the present time, the RDCRN registry is not integrated with other local registries. This ability is being considered for future development. For this reason, we ask that you enter both registries:


What you can do:

  • Join the national registry (see below) in addition to registering with PWSA (USA) at www.pwsausa.org/population.
  • Clinical Trials -  Click here
  • Participate in other research approved by our Scientific Advisory Board and/or our Clinical Advisory Board - Click here

 

 

edited: 02/09/2012

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