Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

NCT04426149 | Completed DMC

• 1 other identifier: UKM PPI/111/8/JEP-2017-826
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

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.

Conditions

Spinocerebellar Ataxia 3

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (2)

• Side effects Profile

  2 monthly intervals for 6 months

• Blood investigation

  At baseline and at 6 months

Study Arms (1)

interventional

EXPERIMENTAL

supplement: trehalose

Dietary Supplement: trehalose

Interventions

trehalose DIETARY_SUPPLEMENT

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

interventional

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• National University of Malaysia: lead

Study Sites (1)

Pusat Perubatan Universiti Kebangsaan Malaysia

Kuala Lumpur, 56000, Malaysia

Location

MeSH Terms

Conditions

Machado-Joseph Disease

Interventions

Trehalose

Condition Hierarchy (Ancestors)

Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Glucans; Polysaccharides; Carbohydrates; Disaccharides; Oligosaccharides; Sugars

Study Officials

• NORLINAH MOHAMED IBRAHIM, MBBCH

  norlinah@ppukm.ukm.edu.my

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 18, 2019
• First Posted: June 11, 2020
• Study Start: March 7, 2018
• Primary Completion: September 7, 2018
• Study Completion: September 7, 2018
• Last Updated: June 11, 2020

Record last verified: 2020-06

Data Sharing

• IPD Sharing: Will not share

Locations

MY (1)