NCT04426149

Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

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

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: National University of Malaysia · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Accepted

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.

Detailed 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.

Conditions

• Spinocerebellar Ataxia 3

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	trehalose	patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily

Timeline

• Start date: 2018-03-07
• Primary completion: 2018-09-07
• Completion: 2018-09-07
• First posted: 2020-06-11
• Last updated: 2020-06-11

Locations

1 site across 1 country: Malaysia