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
Help us to help you!
We need your participation
5202 - Prader-Willi Syndrome and Early-onset Morbid Obesity Natural History
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.
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
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:
email@example.com, Erin L. Youngs, MS: 913-588-1871, email:
firstname.lastname@example.org or Marilyn
Logan, RN: 913-588-1315, email:
University of California at Irvine (ARPWS)
Principal Investigator: Virginia Kimonis, MD
Contact Person: Virginia Kimonis, MD
University of Florida Health Sciences Center (ARPWS)
Principal Investigator: Daniel J. Driscoll, MD, PhD
Contact Person: Christy Hollywood Lynn, RD, LDN
Vanderbilt University Medical Center (ARPWS)
Principal Investigator: Elisabeth Dykens, Ph.D
Contact Person: Elizabeth Roof, M.A., L.P.E.
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
- Clinical Trials -
- Participate in other research approved by our Scientific Advisory
Board and/or our Clinical Advisory Board -