Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

Spinocerebellar Ataxia 3 Clinical Trial

Official title: Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study

Status Completed

Clinical Trial Summary

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.

Clinical Trial Description

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. ;

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia 3
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

• NCT number: NCT04426149
• Study type: Interventional
• Source: National University of Malaysia
• Status: Completed
• Phase: N/A
• Start date: March 7, 2018
• Completion date: September 7, 2018