Spinocerebellar Ataxia 3 Clinical Trial
Official title:
Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study
Verified date | June 2020 |
Source | National University of Malaysia |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
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 |
Country | Name | City | State |
---|---|---|---|
Malaysia | Pusat Perubatan Universiti Kebangsaan Malaysia | Kuala Lumpur |
Lead Sponsor | Collaborator |
---|---|
National University of Malaysia |
Malaysia,
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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