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

Sponsors

Lead Sponsor: National University of Malaysia

Source 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.

Overall Status Completed
Start Date March 7, 2018
Completion Date September 7, 2018
Primary Completion Date September 7, 2018
Phase N/A
Study Type Interventional
Primary Outcome
Measure Time Frame
scale of rating of ataxia (SARA) score months, 2 monthly intervals for 6 months
SCA Functional Index Scores 2 monthly intervals for 6 months
EQ5D3L - quality of life scores 2 monthly intervals for 6 months
Secondary Outcome
Measure Time Frame
Side effects Profile 2 monthly intervals for 6 months
Blood investigation At baseline and at 6 months
Enrollment 13
Condition
Intervention

Intervention Type: Dietary Supplement

Intervention Name: trehalose

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

Arm Group Label: interventional

Eligibility

Criteria:

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

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Last Name Role Affiliation
NORLINAH MOHAMED IBRAHIM, MBBCH Principal Investigator [email protected]
Location
Facility: Pusat Perubatan Universiti Kebangsaan Malaysia
Location Countries

Malaysia

Verification Date

June 2020

Responsible Party

Type: Principal Investigator

Investigator Affiliation: National University of Malaysia

Investigator Full Name: Dr Norlinah Mohamed Ibrahim

Investigator Title: Professor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: interventional

Type: Experimental

Description: supplement: trehalose

Patient Data No
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov