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Fragile X Syndrome clinical trials at UC Health
4 in progress, 3 open to new patients

  • 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

  • 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

  • Measuring thinking skills in children with Fragile X Syndrome

    “Testing cognitive function treatments in Fragile X Syndrome”

    open to eligible people ages 8-18

    Individuals with fragile X syndrome (FXS) demonstrate profound executive function deficits that interfere with learning, socialization and emotion regulation. Extensive research focused on the animal models of FXS show that targeted pharmacological agents can normalize synaptic connectivity and reverse cognitive and behavioral deficits. This translational work has led to multiple national and international controlled trials in humans with FXS now underway. However, in contrast to the heavy focus on medication treatments, there have been no controlled trials to empirically-validate cognitive or behavioral interventions for FXS. The proposed study, the first non-pharmacological controlled trial for FXS, will evaluate the efficacy of Cogmed, a cognitive training program proven to enhance working memory and executive/frontal function in a variety of clinical populations. Demonstration of effective Cogmed training for FXS would represent a major advance in the field, one that may also generalize to other forms of intellectual disability. Furthermore, it is critical to determine whether the targeted pharmacological treatments can accelerate learning and cognitive development. Thus, the validation of Cogmed for FXS will provide a paradigm for testing hypotheses focused on combined efficacy of medication and cognitive training.

    at UC Davis

  • Clinical Study Of caNNabidiol in childrEn and adolesCenTs With Fragile X (CONNECT-FX)

    Sorry, not currently recruiting here

    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

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