Non-Drug Clinical Research

What is a Clinical Study?

A clinical study involves research using human volunteers (also called participants) that is intended to add to medical knowledge. In broad terms, clinical research refers to research done on human subjects with the goal of understanding the natural history of a disease, validating diagnostic tools, or evaluating a treatment or intervention.  There are two main types of clinical studies: clinical trials (also called interventional studies) and observational studies.    In an observational study, investigators assess health outcomes in groups of participants according to a research plan or protocol.  Many researchers that conduct observational studies ask for our help in recruiting for participation.  Sometimes you may be asked to go to their site to participate, and sometimes the protocol may be as simple as completing an online questionnaire.  This type of research is important for understanding many aspects of Rett syndrome.  We will list such studies and request your participation if we review their protocol and know that it is approved by the researcher’s Institutional Review Board (IRB), even if we are not funding the study directly. 

Some current research studies that you may participate in are listed below:

Development of Clinical Guidelines for the Management of Communication in Individuals with Rett Syndrome

We are delighted to announce the start of an international project, funded by a HeART research grant from Rettsyndrome.org, to develop clinical guidelines for the management of communication in individuals with Rett syndrome.

The idea for the project took shape following the third European Rett Syndrome Conference in Maastricht in October 2013. During the next two years the project will be led by the Rett Expertise Centre Netherlands (Gill Townend and prof. dr. Leopold Curfs), with the core work group also including Dr. Theresa Bartolotta (USA), Helena Wandin (Sweden), Anna Urbanowicz (Australia) and Sally-Ann Garrett (UK and Ireland). The project will combine available evidence with expert consensus, following the model used to develop guidelines for the management of scoliosis, nutrition and growth, and most recently bone health, in Rett syndrome.

 

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Rett Expertise Centre Netherlands, GKC
Maastricht University Medical Centre, The Netherlands
Gill Townend
Email: g.townend@maastrichtuniversity.nl

Visual Attention to Print in Children with Rett Syndrome Study

Appalachian State University and the Center for Literacy and Disability Studies at the University of North Carolina announce a new collaborative study of visual attention to print during storybook reading by parents and their children with Rett syndrome. Children, ages 36-96 months (3-8 years), with Rett syndrome and their parents are being recruited to participate in this study.  The entire study is to be conducted online from each participant’s home.

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Appalachian State Univeristy
Dr. David Koppenhaver
Email: koppenhaverd@appstate.edu

Center for Literacy and Disability Studies
Email: clds@unc.edu

Auditory Assessment of Language and Learning

Dr. Sarika Peters at Vanderbilt University seeks enrollment for language/auditory #rettsyndrome and #MECP2-related disorders research project, funded by a Rettsyndrome.org HeART grant. Only one visit, approximatley two hours in length, is required at Vanderbilt in Nashville. A $50 gift card will be offered to each participating family, reduced hotel rates can be arranged as necessary, and free on-campus parking will be extended.

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Vanderbilt University
Dorita Jones, Study Coordinator
Email: dorita.jones@vanderbilt.edu

Experiences of Parents of Children with Special Needs/Disabilities

This study will explore the experiences of parents of children with special needs/disabilities from a multicultural perspective. I hope that this study can help to raise awareness and inform positive change towards the design and use of culturally-sensitive services. Participation in the study consists of taking a 20-minute online survey, is completely confidential. Upon completion of the survey, participants will be eligible to enter a voluntary prize raffle for one of three $100 gift cards to Amazon.

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Washington State University
Erin Okumoto
Email: e.okumoto@email.wsu.edu

IMPROVING UPPER EXTREMITY MOTORSKILLS IN PERSONS WITH RETT SYNDROME USING VIRTUAL REALITY COMPUTER GAMES

The purpose of this study is to facilitate development of or improve purposeful arm movements of individuals with Rett Syndrome using virtual reality as the intervention. Software will convert arm movements into keyboard actions to allow participants to control activities on the computer/tv screen.

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For more information:

If you are in the Baltimore, MD and DC Metro area:
Pam Diener
Email: psd23@georgetown.edu
Phone: (240) 381-1763

If you are in the St. Louis, MO area:
Kourtney Mraz
Email: kmraz@wustl.edu
Phone: (815) 450-0069