Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

NCT ID: NCT04426149

Last Updated: 2020-06-11

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-03-07
• Study Completion Date: 2018-09-07

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.

Conditions

Spinocerebellar Ataxia 3

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

interventional

supplement: trehalose

Group Type: EXPERIMENTAL

trehalose

Intervention Type: DIETARY_SUPPLEMENT

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

Interventions

trehalose

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

Intervention Type: DIETARY_SUPPLEMENT

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

National University of Malaysia

OTHER

Sponsor Role: lead

Responsible Party

Dr Norlinah Mohamed Ibrahim

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

NORLINAH MOHAMED IBRAHIM, MBBCH

Role: PRINCIPAL_INVESTIGATOR

norlinah@ppukm.ukm.edu.my

Locations

Pusat Perubatan Universiti Kebangsaan Malaysia

Kuala Lumpur, , Malaysia

Countries

Malaysia

Other Identifiers

UKM PPI/111/8/JEP-2017-826

Identifier Type: -

Identifier Source: org_study_id