Status:
UNKNOWN
Clinical Trial Readiness for SCA1 and SCA3
Lead Sponsor:
The Methodist Hospital Research Institute
Collaborating Sponsors:
University of Michigan
University of Minnesota
Conditions:
Spinocerebellar Ataxia Type 1
Spinocerebellar Ataxia 3
Eligibility:
All Genders
18-65 years
Brief Summary
The investigators plan to fill the gap between the current state of clinical trial readiness and the optimal one for SCA1 and SCA3, which are fatal rare diseases with no treatments. Through US-Europea...
Detailed Description
Spinocerebellar ataxia types 1 (SCA1) and 3 (SCA3) are rare, inherited neurodegenerative disorders that relentlessly progress to total disability and death. SCA1 is the fastest progressing SCA while S...
Eligibility Criteria
Inclusion
- Signed informed consent (no study-related procedures may be performed before the subject has signed the consent form).
- Subjects of either sex aged 18 to 65 with presence of symptomatic ataxic disease or asymptomatic mutation carrier or
- Subjects with definite molecular diagnosis of SCA1 or SCA3 or another affected family member
- Subjects of any age with previous diagnosis of Early stage SCA1 and SCA3
- Subjects capable of understanding and complying with protocol requirements
- No changes in physical/occupational therapy status within two months prior to enrollment
Exclusion
- Subjects currently receiving, or having received within 2 months prior to enrollment into this study, any investigational drug.
- Subjects who do not wish to or cannot comply with study procedures.
- Genotype consistent with other inherited ataxias
- Changes in coordinative physical and occupational therapy for ataxia 2 months prior to study participation
- Concomitant disorder(s) or condition(s) that affects assessment of ataxia or severity of ataxia during this study
- AIM 2 exclusion criteria also includes the inability to undergo MRI scanning and weight over 300lbs, presence of structural abnormalities such as subdural hematoma or primary or metastatic neoplasms and concurrent illnesses or treatment interfering with cognitive function such as stroke or normal pressure hydrocephalus.
Key Trial Info
Start Date :
August 16 2018
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 31 2023
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT03487367
Start Date
August 16 2018
End Date
December 31 2023
Last Update
January 19 2022
Active Locations (1)
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1
Houston Methodist Hospital
Houston, Texas, United States, 77030