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Rett Syndrome | Overview

Phase 2 Clinical Trial for the Treatment of Rett Syndrome

  • P.I. Charles Nelson, PhD and Mustafa Sahin, PhD
  • Participants: This study is enrolling children with Rett syndrome between 2-10 years old. Participants must have a clinical diagnosis of "Classic" or "Typical" Rett syndrome and a documented genetic MECP2 mutation. Subjects must also be in a "stable" stage of the syndrome, i.e. no longer experiencing symptoms or signs of regression (loss of skill).

Phase 2 is a double-blinded, placebo-controlled cross-over study designed to test the efficacy of IGF-1 for treatment of Rett syndrome. The Nelson Lab uses both EEG and a cognitive developmental assessment to evaluate changes in cortical function across treatment. For the EEG, we do this by evaluating two well characterized systems in the brain (the auditory system and the visual system). We use traditional ERP paradigms as well as steady-state presentation of auditory and visual stimuli and power spectra analysis to assess cortical function of V1 and auditory cortex. In addition, the Nelson Lab has adapted the Mullen Scales of Early Learning for use with children with Rett syndrome.

For full participation details, contact us via e-mail or at 857-218-3011.