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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| interventional | Experimental | supplement: trehalose |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| trehalose | Dietary Supplement | patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily |
|
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| NORLINAH MOHAMED IBRAHIM, MBBCH | norlinah@ppukm.ukm.edu.my | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Pusat Perubatan Universiti Kebangsaan Malaysia | Kuala Lumpur | 56000 | Malaysia |
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| ID | Term |
|---|---|
| D017827 | Machado-Joseph Disease |
| ID | Term |
|---|---|
| D020754 | Spinocerebellar Ataxias |
| D002524 | Cerebellar Ataxia |
| D002526 | Cerebellar Diseases |
| D001927 | Brain Diseases |
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| ID | Term |
|---|---|
| D014199 | Trehalose |
| ID | Term |
|---|---|
| D005936 | Glucans |
| D011134 | Polysaccharides |
| D002241 | Carbohydrates |
| D004187 | Disaccharides |
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| At baseline and at 6 months |
| D002493 |
| Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013132 | Spinocerebellar Degenerations |
| D013118 | Spinal Cord Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D001259 | Ataxia |
| D020820 | Dyskinesias |
| D009461 | Neurologic Manifestations |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009844 |
| Oligosaccharides |
| D000073893 | Sugars |