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Rett Syndrome clinical trials at University of California Health

2 in progress, 1 open to eligible people

Showing trials for
  • Rett Syndrome Registry

    open to eligible people ages 0-99

    The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with the IRSF Rett Syndrome Center of Excellence Network medical directors, this study collects data on the signs and symptoms of Rett syndrome as reported by the Rett syndrome experts and by the caregivers of individuals with Rett syndrome. This study will be used to develop consensus based guidelines for the care of your loved ones with Rett syndrome and to facilitate the development of better clinical trials and other aspects of the drug development path for Rett syndrome.

    at UCSF

  • TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

    Sorry, not currently recruiting here

    The REVEAL Pediatric Study is a multi-center, Phase 1/2 open-label, dose-escalation and dose-expansion study of TSHA-102, an investigational gene therapy, in pediatric females with Rett Syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is up to 6 years.

    at UCSD

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