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

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.

Study Overview

• Status: Completed
• Conditions: Spinocerebellar Ataxia 3
• Intervention / Treatment: Dietary supplement: trehalose

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 Type: Interventional
• Enrollment (Actual): 13
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Malaysia
  • Kuala Lumpur, Malaysia, 56000
    • Pusat Perubatan Universiti Kebangsaan Malaysia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: interventional; supplement: trehalose	Dietary supplement: trehalose; patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: National University of Malaysia
• Investigators: Principal Investigator: NORLINAH MOHAMED IBRAHIM, MBBCH, norlinah@ppukm.ukm.edu.my

Study record dates

Study Major Dates

• Study Start (Actual): March 7, 2018
• Primary Completion (Actual): September 7, 2018
• Study Completion (Actual): September 7, 2018

Study Registration Dates

• First Submitted: April 18, 2019
• First Submitted That Met QC Criteria: June 8, 2020
• First Posted (Actual): June 11, 2020

Study Record Updates

• Last Update Posted (Actual): June 11, 2020
• Last Update Submitted That Met QC Criteria: June 8, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Genetic Diseases, Inborn; Neurodegenerative Diseases; Dyskinesias; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Cerebellar Diseases; Ataxia; Cerebellar Ataxia; Spinocerebellar Ataxias; Spinocerebellar Degenerations; Machado-Joseph Disease
• Other Study ID Numbers: UKM PPI/111/8/JEP-2017-826

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No