Rett Syndrome clinical trials at UCSF

2 research studies open to eligible people

Rett syndrome is a rare neurodevelopmental disorder that impairs movement, speech, and hand use. UCSF is testing an investigational gene therapy to monitor safety and effects on symptoms. UCSF is maintaining a registry that gathers long-term health and genetic information.

Showing trials for

  • Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

    open to eligible females ages 3 years and up

    This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.

    Oakland, California and other locations

  • 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.

    Oakland, California and other locations

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