Spinocerebellar Ataxia Type 3NCT04399265
Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3
A study testing Trehalose and Maltose in people with Spinocerebellar Ataxia Type 3, run by National University of Malaysia at 1 site in Malaysia.
Trial summary
This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3. A total of 40 participants with SCA3 will be recruited, with 20 participants to be administered with trehalose while another 20 participants to be administered with a… Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Spinocerebellar Ataxia Type 3
- What is being tested?
- Trehalose and Maltose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteMalaysia
- Study dates
- 24 Feb 2020to 30 Jun 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age between 18 and 85 years old.
- 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.
Who cannot join
- Diagnosis of Diabetes Mellitus Type 1 or Type 2
- 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.
Trial locations and contacts
1 site in 1 country.
Malaysia1 site, 1 recruiting
- Recruiting
UKM Medical Centre
Cheras, Kuala Lumpur, 56000
Kah Hui Yap, Master, 0165536721, kahhui0411@gmail.com
Norlinah Mohamed Ibrahim, MRCP, +60122145306, norlinah@ppukm.ukm.edu.my
About this study
Official title: A Randomised Controlled Trial on the Clinical Efficacy of Oral Trehalose in Patients With Spinocerebellar Ataxia 3: Clinical & FMRI Correlation
This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3. A total of 40 participants with SCA3 will be recruited, with 20 participants to be administered with trehalose while another 20 participants to be administered with a maltose placebo.
Read the detailed description
Spinocerebellar ataxia 3 (SCA3) is a rare form of inherited neurodegenerative disease involving progressive degeneration of spinocerebellar tract. SCA3 is characterised by increasingly worsening cerebellar function leading to gait abnormalities and poor coordination, dysarthria, and abnormal eye movements. Non-ataxia features include pyramidal and extrapyramidal manifestations, sensorimotor, neuropsychological and psychiatric symptoms. This is attributed to the role of cerebellum in motor, cognitive and affective processing (i.e. cerebellar cognitive and affective syndrome; CCAS), as well as its extensive connection with cerebral structures.
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
- Spinocerebellar Ataxia Type 3
- Being tested
- Trehalose, Maltose
- Keywords
- Spinocerebellar ataxia Type 3, Cerebellar Cognitive and Affective Syndrome, Trehalose, Neuropsychology, Magnetic resonance imaging
- Medical subject headings
- Machado-Joseph Disease, Cerebellar Cognitive Affective Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrehaloseDietary supplement
A disaccharide sugar usually used as food preservative, but for this study it is given at a higher dose
MaltoseDietary supplement
Maltose power
| Study group | Role | What they receive |
|---|---|---|
| Trehalose | Experimental | Trehalose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months. Receives: Dietary Supplement: Trehalose |
| Maltose placebo | Placebo comparator | Isocaloric maltose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months. Receives: Dietary Supplement: Maltose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes from Baseline Scale for Ataxia Rating Assessment (SARA) at 3 months, 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
An 8-item scale to quantify the severity of ataxia with a scoring of 0 (no ataxia) to 40 (most severe ataxia). Total time taken for test administration is estimated at 10 minutes.
Measured: Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.
Secondary outcomes (8)
Changes from Baseline Spinocerebellar Ataxia Functional Index (SCAFI) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
A validated multi-modal assessment tool that is composed of: a) timed 8 metre walk (8MW); b) the 9-hole peg test (9HPT); and c) the rate of "PATA" repetition over 10 seconds (PATA) to rate speech performance. Total time taken for test administration is estimated at 10 minutes.
Measured: Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Inventory of Non-Ataxia Symptoms (INAS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
A validated scale assessing non-cerebellar signs with a scoring of 0 (no non-ataxia sign) to 16 (all assessed systems affected). Total time taken for test administration is estimated at 10 minutes.
Measured: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Cerebellar Cognitive & Affective Syndrome (CCAS) Scale at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
CCAS Scale is a brief cognitive screening tool to help identify CCAS in patients with cerebellar impairment. It derives a total score of 120 as a continuous measure; as well as an ordinal measure in accordance to the number of failed tests: 1) Possible CCAS = 1 failed test; 2) Probable CCAS = 2 failed tests; Definite CCAS = 3 or more failed tests. Four different forms were available to minimize the practice effect. Total time taken for test administration is estimated at 10 minutes.
Measured: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Wechsler Adult Intelligence Scale (WAIS - 4) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
WAIS - 4 is an IQ test designed to measure intelligence and cognitive ability in adults and older adolescents. The selected subtests are Matrix Reasoning, Digit Span, and Coding. These subtests are used to measure abstract reasoning, WM, and processing speed respectively. These index and subtests have good reliability
Measured: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Delis-Kaplan Executive Function System (D - KEFS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
D - KEFS is a neuropsychological battery designed to measure various subdomains of executive function from 8 - 89 years old. The selected subtests are: Tower Test, Trail Making Test (TMT), Colour-Word Interference Test (CWIT). These tests measure planning, set-shifting, and inhibition abilities respectively.
Measured: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Repeatable Battery for the Assessment of Neuropsychological Status Update (RBANS Update) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
RBANS Update is a neuropsychological battery designed to measure 5 neuropsychological domains from 12:0 - 89:11 years old. The selected subtests are: Figure Copy and Figure Recall. These tests measure visual construction, visual memory, and language abilities respectively.
Measured: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Resting state fMRI at 3 months.
The level of measurement of oxy-Hb (activation level) is continuous; greater oxy-Hb (mM.mm) indicates greater activation.
Measured: Baseline and 3 months
Changes from Magnetic resonance spectroscopy at 3 months.
The level of measurement of N-Acetyl Aspartate (NAA; metabolism) is continuous; greater NAA (ppm) indicates greater metabolism.
Measured: Baseline and 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2020
- Study start24 Feb 2020
- Primary completion30 May 2023 (estimated)
- Study completion30 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Type 3Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study will employ a double-blind randomised controlled trial (RCT) with two arms. Patients with SCA3 will be randomly allocated to oral trehalose group and a maltose placebo group. A total of 20 participants will be allocated to each group. There is a possibility of including other types of SCA at later stage of the study.
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National University of Malaysia
- Sponsor type
- University, hospital or other
- Collaborators
- Radboud University Medical Center
- Responsible party
- Principal investigator, Dr Norlinah Mohamed Ibrahim, National University of Malaysia
- Investigators
- Norlinah Mohamed Ibrahim, MRCP, Principal investigator, National University of Malaysia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04399265
- Sponsor's protocol ID
- DIP-2019-007
- Status last verified
- Aug 2022
- First submitted
- 11 Mar 2020
- Last updated
- 16 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomised Controlled Trial on the Clinical Efficacy of Oral Trehalose in Patients With Spinocerebellar Ataxia 3: Clinical & FMRI Correlation
- ClinicalTrials.gov ID
- NCT04399265
- Acronym / sponsor ID
- DIP-2019-007
- Conditions
- Spinocerebellar Ataxia Type 3
- Keywords
- Spinocerebellar ataxia Type 3, Cerebellar Cognitive and Affective Syndrome, Trehalose, Neuropsychology, Magnetic resonance imaging
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- National University of MalaysiaUniversity, hospital or other
- Collaborators
- Radboud University Medical Center
- Study start
- 24 Feb 2020 (actual)
- Primary completion
- 30 May 2023 (estimated)
- Study completion
- 30 Jun 2023 (estimated)
- First posted
- 22 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates the effectiveness of oral trehalose in alleviating the neuropathological and motor behaviour deficits among patients with SCA3. A total of 40 participants with SCA3 will be recruited, with 20 participants to be administered with trehalose while another 20 participants to be administered with a maltose placebo.
Detailed description
Spinocerebellar ataxia 3 (SCA3) is a rare form of inherited neurodegenerative disease involving progressive degeneration of spinocerebellar tract. SCA3 is characterised by increasingly worsening cerebellar function leading to gait abnormalities and poor coordination, dysarthria, and abnormal eye movements. Non-ataxia features include pyramidal and extrapyramidal manifestations, sensorimotor, neuropsychological and psychiatric symptoms. This is attributed to the role of cerebellum in motor, cognitive and affective processing (i.e. cerebellar cognitive and affective syndrome; CCAS), as well as its extensive connection with cerebral structures.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
- Model description
- This study will employ a double-blind randomised controlled trial (RCT) with two arms. Patients with SCA3 will be randomly allocated to oral trehalose group and a maltose placebo group. A total of 20 participants will be allocated to each group. There is a possibility of including other types of SCA at later stage of the study.
| Arm | Type | Receives |
|---|---|---|
| Trehalose | Experimental | Dietary Supplement: TrehaloseTrehalose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months. |
| Maltose placebo | Placebo comparator | Dietary Supplement: MaltoseIsocaloric maltose powder form to be dissolved in water, to be consumed by mouth, every day for 3 months. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age between 18 and 85 years old.
- 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.
Key exclusion criteria (6 listed)
- Diagnosis of Diabetes Mellitus Type 1 or Type 2
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TrehaloseA disaccharide sugar usually used as food preservative, but for this study it is given at a higher dose | Dietary supplement | Trehalose |
| MaltoseMaltose power | Dietary supplement | Maltose placebo |
Primary outcome
Changes from Baseline Scale for Ataxia Rating Assessment (SARA) at 3 months, 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.
An 8-item scale to quantify the severity of ataxia with a scoring of 0 (no ataxia) to 40 (most severe ataxia). Total time taken for test administration is estimated at 10 minutes.
8 secondary outcomes
Changes from Baseline Spinocerebellar Ataxia Functional Index (SCAFI) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Inventory of Non-Ataxia Symptoms (INAS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Cerebellar Cognitive & Affective Syndrome (CCAS) Scale at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Wechsler Adult Intelligence Scale (WAIS - 4) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Delis-Kaplan Executive Function System (D - KEFS) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Repeatable Battery for the Assessment of Neuropsychological Status Update (RBANS Update) at 3 months and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Time frame: Baseline, 3 months, and 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up.
Changes from Baseline Resting state fMRI at 3 months.
Time frame: Baseline and 3 months
Changes from Magnetic resonance spectroscopy at 3 months.
Time frame: Baseline and 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Norlinah Mohamed Ibrahim, MRCP principal investigator
National University of Malaysia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Trehalose and Maltose in people with Spinocerebellar Ataxia Type 3, run by National University of Malaysia at 1 site in Malaysia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Malaysia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Kah Hui Yap, Master (+60165536721, kahhui0411@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes from Baseline Scale for Ataxia Rating Assessment (SARA) at 3 months, 6 months; as well as 3, 6, 9, and 12 months post-treatment follow-up. (time frame: Baseline, 3 months, and 6 months, as well as 3, 6, 9, and 12 months post-treatment follow-up.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Aug 2022.