Machado–Joseph Disease clinical trials at UCSF
2 in progress, 1 open to eligible people
Machado-Joseph Disease is a genetic illness that leads to movement and coordination problems. UCSF is conducting research to track the disease's progression and identify genetic changes that affect it. The studies aim to better understand balance and speech challenges in affected individuals.
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 Michael Geschwind, MD.
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