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Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

Primary Purpose

Spinocerebellar Ataxia 3

Status

Completed

Phase

Not Applicable

Locations

Malaysia

Study Type

Interventional

Intervention

trehalose

About this trial

This is an interventional treatment trial for Spinocerebellar Ataxia 3

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

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

Exclusion Criteria:

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

Sites / Locations

Pusat Perubatan Universiti Kebangsaan Malaysia

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

interventional

Arm Description

supplement: trehalose

Outcomes

Primary Outcome Measures

scale of rating of ataxia (SARA) score months,

Assessment of SARA scores by a single assessor

SCA Functional Index Scores

Assessment of SCAFI by a single assessor

EQ5D3L - quality of life scores

Assessment of quality of life scores

Secondary Outcome Measures

Side effects Profile

Adverse Effects

Blood investigation

Measurement of renal profile, fasting blood glucose, full blood count and liver profile

Full Information

NCT ID

NCT04426149

First Posted

April 18, 2019

Last Updated

June 8, 2020

Sponsor

National University of Malaysia

1. Study Identification

Unique Protocol Identification Number

NCT04426149

Brief Title

Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

June 2020

Overall Recruitment Status

Completed

Study Start Date

March 7, 2018 (Actual)

Primary Completion Date

September 7, 2018 (Actual)

Study Completion Date

September 7, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

National University of Malaysia

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Product Manufactured in and Exported from the U.S.

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Spinocerebellar Ataxia 3

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

interventional

Arm Type

Experimental

Arm Description

supplement: trehalose

Intervention Type

Dietary Supplement

Intervention Name(s)

trehalose

Intervention Description

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

Primary Outcome Measure Information:

Title

scale of rating of ataxia (SARA) score months,

Description

Assessment of SARA scores by a single assessor

Time Frame

2 monthly intervals for 6 months

Title

SCA Functional Index Scores

Description

Assessment of SCAFI by a single assessor

Time Frame

2 monthly intervals for 6 months

Title

EQ5D3L - quality of life scores

Description

Assessment of quality of life scores

Time Frame

2 monthly intervals for 6 months

Secondary Outcome Measure Information:

Title

Side effects Profile

Description

Adverse Effects

Time Frame

2 monthly intervals for 6 months

Title

Blood investigation

Description

Measurement of renal profile, fasting blood glucose, full blood count and liver profile

Time Frame

At baseline and at 6 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s) Consent to participate in the study The age of 18 years and older Exclusion Criteria: Unconfirmed SCA 3 Concomitant disorder(s) that affect SARA and other ataxia measures used in this study Diabetes Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of absorption results in diarrhoea and intestinal distress. Less than 18 years old

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

NORLINAH MOHAMED IBRAHIM, MBBCH

Organizational Affiliation

norlinah@ppukm.ukm.edu.my

Official's Role

Principal Investigator

Facility Information:

Facility Name

Pusat Perubatan Universiti Kebangsaan Malaysia

City

Kuala Lumpur

ZIP/Postal Code

56000

Country

Malaysia

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

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