Spinocerebellar Ataxia Type 27B Natural History Study (SCA27B-NHS)
Recruiting
Observational Study
Spinocerebellar Ataxia Type 27B
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 18 – 99
- Sex
- Any
- Study type
- Observational
- Participants needed
- 300 (estimated)
- Sponsor
- University Hospital Tuebingen · Other
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About This Trial
This international, multi-center, multi-modal, and prospective observational cohort study aims to validate trial outcomes for capturing disease progression in Spinocerebellar Ataxia Type 27B (SCA27B), with combined multi-modal capture of clinical outcome assessments, digital-motor assessments, and molecular biomarkers.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* SCA27B: genetic diagnosis of ≥250 uninterrupted GAA repeat expansions in FGF14
* SCA27B risk subject: asymptomatic first-degree relative of SCA27B participant with known or unknown carrier status
* Unrelated healthy controls: no signs or history of neurological or psychiatric …
Contacts