Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

Not Applicable

Completed

• Conditions: Spinocerebellar Ataxia 3
• Interventions: Dietary Supplement: trehalose

• Registration Number: NCT04426149
• Lead Sponsor: National University of Malaysia

Brief 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.

Study Timeline

• Study Start: 2018-03-07
• Primary Completion: 2018-09-07
• Study Completion: 2018-09-07
• Study First Submitted: 2019-04-18
• Status Verified: 2020-06
• Study First Submitted QC: 2020-06-08
• Last Update Submitted: 2020-06-08
• Study First Posted: 2020-06-11
• Last Update Posted: 2020-06-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

Inclusion Criteria

1. DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s)
2. Consent to participate in the study
3. The age of 18 years and older

Exclusion Criteria

1. Unconfirmed SCA 3
2. Concomitant disorder(s) that affect SARA and other ataxia measures used in this study
3. Diabetes
4. Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of absorption results in diarrhoea and intestinal distress.
5. Less than 18 years old

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
interventional	trehalose	supplement: trehalose

Primary Outcome Measures

Name	Time	Method
scale of rating of ataxia (SARA) score months,	2 monthly intervals for 6 months	Assessment of SARA scores by a single assessor
SCA Functional Index Scores	2 monthly intervals for 6 months	Assessment of SCAFI by a single assessor
EQ5D3L - quality of life scores	2 monthly intervals for 6 months	Assessment of quality of life scores

Secondary Outcome Measures

Name	Time	Method
Blood investigation	At baseline and at 6 months	Measurement of renal profile, fasting blood glucose, full blood count and liver profile
Side effects Profile	2 monthly intervals for 6 months	Adverse Effects

Trial Locations

Locations (1)

Pusat Perubatan Universiti Kebangsaan Malaysia; 🇲🇾 Kuala Lumpur, Malaysia