Spinocerebellar Ataxia Type 3 Clinical Trial
Official title:
Clinical Research Consortium for Spinocerebellar Ataxias (CRC-SCA) to Study Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias (SCA)
Verified date | May 2023 |
Source | University of Florida |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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.
Status | Recruiting |
Enrollment | 800 |
Est. completion date | May 19, 2024 |
Est. primary completion date | May 19, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Years and older |
Eligibility | Inclusion Criteria: - Presence of symptomatic ataxic disease - Definite molecular diagnosis of SCA 1, 2,3,or 6 either in the subject or another affected family member - Willingness to participate in the study and ability to give informed consent. - Age 6 years and above Exclusion Criteria: - Known recessive, X-linked and mitochondrial ataxias - Exclusion of SCA 1, 2, 3 and 6 by previous DNA testing, - A lack of willingness to participate in the study |
Country | Name | City | State |
---|---|---|---|
United States | University of Michigan | Ann Arbor | Michigan |
United States | Emory University | Atlanta | Georgia |
United States | John Hopkins University | Baltimore | Maryland |
United States | Harvard University | Boston | Massachusetts |
United States | University of Chicago | Chicago | Illinois |
United States | University of Florida | Gainesville | Florida |
United States | University of California Los Angeles | Los Angeles | California |
United States | University of Minnesota | Minneapolis | Minnesota |
United States | Columbia University | New York | New York |
United States | University of Utah | Salt Lake City | Utah |
United States | University of California San Francisco | San Francisco | California |
United States | University of South Florida | Tampa | Florida |
Lead Sponsor | Collaborator |
---|---|
University of Florida | National Ataxia Foundation, University of California, Los Angeles |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The disease's progression over time using clinical rating scales and timed performance measures. | Indefinitely (for as long as the study is open and you wish to participate) | ||
Primary | Relation between the genetic modifiers and the age at onset of disease and disease progression rates. | Indefinitely (for as long as the study is open and you wish to participate) | ||
Secondary | The effects of the disease on the Activities of Daily Living (ADL)in patients with Spinocerebellar Ataxias | indefinitely |
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