Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

Spinocerebellar Ataxia 3 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04426149
• Other study ID #: UKM PPI/111/8/JEP-2017-826
• Status: Completed
• Phase: N/A
• Start date: March 7, 2018
• Est. completion date: September 7, 2018

Study information

• Verified date: June 2020
• Source: National University of Malaysia
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: September 7, 2018
• Est. primary completion date: September 7, 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia 3
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

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

Locations

Country	Name	City	State
Malaysia	Pusat Perubatan Universiti Kebangsaan Malaysia	Kuala Lumpur

Sponsors (1)

Lead Sponsor	Collaborator
National University of Malaysia

Country where clinical trial is conducted

Malaysia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	scale of rating of ataxia (SARA) score months,	Assessment of SARA scores by a single assessor	2 monthly intervals for 6 months
Primary	SCA Functional Index Scores	Assessment of SCAFI by a single assessor	2 monthly intervals for 6 months
Primary	EQ5D3L - quality of life scores	Assessment of quality of life scores	2 monthly intervals for 6 months
Secondary	Side effects Profile	Adverse Effects	2 monthly intervals for 6 months
Secondary	Blood investigation	Measurement of renal profile, fasting blood glucose, full blood count and liver profile	At baseline and at 6 months