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Fragile X Syndrome clinical trials at UC Health
5 research studies open to eligible people

  • A Study Of Experimental Cannabidiol in Children and Adolenscents With Fragile X Syndrome (CONNECT-FX Study)

    open to eligible people ages 3-17

    This study will evaluate the efficacy and safety of ZYN002, a clear cannabidiol (CBD) gel that can be applied to the skin (called transdermal application) twice a day for the treatment of behavioral symptoms of Fragile X Syndrome (FXS). Eligible participants will then participate in up to a 14 week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to < 18 years, will be eligible to participate.

    at UC Davis

  • A Study of Experimental Treatment With Metformin for Fragile X Syndrome

    “Learn if Metformin may be helpful for language, behavior problems, and obesity / excessive appetite in patients with Fragile X Syndrome.”

    open to eligible people ages 6-25

    This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 25 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a series of tests. The primary objectives are to assess safety, tolerability, and efficacy of metformin in the treatment of language deficits, behavior problems, and obesity/excessive appetite in individuals with fragile X syndrome.

    at UC Davis

  • A Study of the Experimental Medicine OV101 For Fragile X Syndrome

    open to eligible males ages 13-22

    The purpose of this study is to assess the safety, tolerability and efficacy of oral OV101 (gaboxadol) in subjects with Fragile X syndrome.

    at UC Davis

  • A Study of the Long-Term Safety and Tolerability of Experimental ZYN002 in Children and Adolescents With Fragile X Syndrome (FXS)

    open to eligible people ages 3-18

    ZYN002 is a pharmaceutically manufactured Cannabidiol (CBD) that is developed as a clear gel that can be applied to the skin (called transdermal delivery). The gel will be applied to clean, dry, intact skin of the shoulders and/or upper arms. Only participants from the ZYN2-CL-016 study who meet the inclusion criteria and none of the exclusion criteria for study ZYN2-CL-017 are eligible. Parents/caregivers will apply the study gel twice daily for the 52-week treatment period.

    at UC Davis

  • AFQ056 for Language Learning in Children With FXS

    “If your child is between the ages of 32 months and 6 years and has fragile X syndrome, we invite them to take part in this study.”

    open to eligible people ages 32 months to 6 years

    The overall goals are to change the paradigm for development of mechanism targeted pharmacotherapy in neurodevelopmental disorders and provide a definitive test of the mGluR theory in humans by determining whether AFQ056, an mGluR5 negative modulator, can enhance neural plasticity in the form of language learning during an intensive language intervention in very young children with fragile X syndrome. This trial therefore will use an innovative but exploratory new trial design to develop a different way to examine efficacy of an agent with substantial support as a drug targeting CNS plasticity in preclinical models of a developmental disorder. If the design is successful, this trial can serve as a model for future trials of mechanistically-targeted treatments operating on neural plasticity in other neurodevelopmental disorders.

    at UC Davis

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