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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04426149
Other study ID # UKM PPI/111/8/JEP-2017-826
Secondary ID
Status Completed
Phase N/A
First received
Last updated
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
Health authority
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

Study Design


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
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