Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
NA
20 participants
INTERVENTIONAL
2020-02-24
2023-06-30
Brief Summary
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Detailed Description
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Trehalose is an omnipotent disaccharide molecule found in lower and higher life forms except in vertebrates. It has an amorphous (i.e. non-reducing) property, which is shown in its high hydrophilicity, chemical stability and strong resistance to denaturation / breakdown by heat, acid or enzyme. It is also shown to help refold partially denatured protein, thereby stabilizes protein aggregates, including those of polyglutamine, in vivo as well as in vitro. This has provided an avenue in which trehalose as a therapeutic agent for neurodegenerative disorders with pathological changes of protein aggregates.
In this study, a double-blinded randomised controlled trial (RCT) will be employed. A total of 40 patients with SCA3 will be randomly allocated to oral trehalose group and a placebo group (20 participants for each arm). With regards to clinical outcomes, motor and cognitive performances will be assessed to infer the efficacy of trehalose. Likewise, structural, resting-state fMRI (i.e. functional connectivity), and MR spectroscopy (i.e. metabolism), will be used as imaging biomarkers in this study.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Trehalose
Trehalose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months.
Trehalose
A disaccharide sugar usually used as food preservative, but for this study it is given at a higher dose
Maltose placebo
Isocaloric maltose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months.
Maltose
Maltose power
Interventions
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Trehalose
A disaccharide sugar usually used as food preservative, but for this study it is given at a higher dose
Maltose
Maltose power
Eligibility Criteria
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Inclusion Criteria
* Genetically confirmed SCA 3 diagnosis.
* Presence of progressive cerebellar syndrome (i.e. symptomatic) and genetic confirmation of SCA 3 in one of the immediate family members with similar clinical syndrome.
* Able to read, speak, and understand English or Malay.
Exclusion Criteria
* Presence of any concomitant neurological condition that might interfere with clinical measures used in this study.
* Presence of contraindication or hypersensitivity to trehalose.
* Use of stimulant / medication, caffeine, and tobacco within less than 24 hours washout period prior to assessment and / or scan.
* Presence of severe visual and / or auditory perceptual deficits.
* MRI contra-indications: claustrophobia, pregnancy, electronic implants (e.g. pacemaker) in the body, aneurysm clip, and current or past employment as machinists, welders or metal workers.
18 Years
85 Years
ALL
No
Sponsors
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Radboud University Medical Center
OTHER
National University of Malaysia
OTHER
Responsible Party
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Dr Norlinah Mohamed Ibrahim
Professor
Principal Investigators
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Norlinah Mohamed Ibrahim, MRCP
Role: PRINCIPAL_INVESTIGATOR
National University of Malaysia
Locations
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UKM Medical Centre
Cheras, Kuala Lumpur, Malaysia
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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DIP-2019-007
Identifier Type: -
Identifier Source: org_study_id
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