5214: Development of a Behavioral Outcome Measure for Rett Syndrome (RettBe)

The purpose of this study is to collect more information about behaviors in children with RTT. The results from this questionnaire will be used to develop a questionnaire which will be called RettBe. The goal is that RettBe will help doctors and researchers evaluate behaviors in children with Rett Syndrome, so they can find more effective ways to manage these behaviors.

For Diseases:

  • Rett Syndrome


It has been recently recognized that girls and women with RTT display a wide range of behavioral problems and that these can substantially affect their quality of life. However, management of these abnormal behaviors requires appropriate diagnosis and characterization of these behaviors. The scales that are currently in use are limited in their sensitivity and selectivity for common behaviors seen in children with RTT. With an assessment that can clearly measure current and past behavior across development, researchers may be able to find more effective ways to manage clinically relevant and problematic behaviors in patients with RTT.

This is an observational study that involves collecting information through an on-line questionnaire about behavior.

Targeted Enrollment

To be eligible to participate, you must:

Be a female between the age of 3-18. Participants are expected to already have had and be able to provide result of their gene testing of MECP2.


  • Be diagnosed with Rett Syndrome
  • Have Post-regression status
  • Be enrolled in the Rett Syndrome, MECP2 Duplication and Rett-Related Disorders Natural History Study (RTT5211)
  • Be English speaking

You are not eligible to participate if:

  • You do not meet the above criteria.

In order to participate in a study, you must personally contact the study coordinator.

Miranda Williams
Phone: 813-396-9567
Email: Miranda.Williams@epi.usf.edu