Machado–Joseph Disease clinical trials at UCSF

2 in progress, 1 open to eligible people

Machado-Joseph Disease is a hereditary disorder that impacts the brain and movement. At UCSF, researchers are investigating how this disease changes over time. They are also studying genetic influences on issues like balance and speech.

Showing trials for

  • Genetic Modifiers in Spinocerebellar Ataxias

    open to eligible people ages 6 years and up

    Spinocerebellar ataxias (SCA) are genetic neurological diseases that cause imbalance, poor coordination, and speech difficulties. There are different kinds of SCA and this study will focus on types 1, 2,3, and 6 (SCA 1, SCA 2, SCA 3 , also known as Machado-Joseph disease and SCA 6). The diseases are rare, slowly progressive, cause increasingly severe neurological difficulties and are variable across and within genotypes. The purpose of this research study is to bring together a group of experts in the field of SCA for the purpose of learning more about the disease. The research questions are: 1. How does your disease progress over time? 2. What are the best ways to measure the progression? 3. Do some genes, other than the gene that is abnormal in your disease, have any effect on the way the disease behaves? This is a nationwide study and we expect that 800 patients will participate all over the USA. The participants will be in the study for an indeterminate period of time. Study visits will be done every 6 or 12 months depending on the participating site.

    San Francisco, California and other locations

  • Troriluzole in Adult Participants With Spinocerebellar Ataxia

    Sorry, in progress, not accepting new patients

    The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

    San Francisco, California and other locations

Our lead scientists for Machado–Joseph Disease research studies include .

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