Spinocerebellar Ataxia 3 Clinical Trial
Official title:
Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study
There are no clinically established treatments which have been proven to delay the disease
progression in spinocerebellar ataxia (SCA) 3. Most available treatments are only for symptom
alleviation, and thus the majority of patients will eventually progress to needing and wheel
chair and eventually bedridden.
As trehalose appear to be potentially promising treatment in SCA, the investigators aim to
conduct this study using oral trehalose in our genetically confirmed SCA 3 patients.
This prospective single arm interventional study involved 13 genetically confirmed spinocerebellar ataxia (SCA) 3 patients with no concomitant diabetes, over 6 months. Following baseline assessment, patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily. Assessments were performed at baseline, 2, 4 and 6 months using ataxia rating scales (SARA, SCAFI and INAS) and EQ-5D-3L scale for quality of life assessment. ;
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