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Machado–Joseph Disease clinical trials at UCSF

3 in progress, 2 open to eligible people

Showing trials for
  • A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

    open to eligible people ages 18-70

    The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3). The secondary objective of this study is to characterize the multiple-dose pharmacokinetics (PK) of BIIB132 administered via IT injection to participants with SCA3.

    San Francisco, California and other locations

  • Natural History Study of and 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 Subjects With Spinocerebellar Ataxia

    Sorry, in progress, not accepting new patients

    The purpose of this study is to compare the efficacy of Troriluzole (200mg 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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