Spinocerebellar Ataxia 3NCT04426149
Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3
A study testing trehalose in people with Spinocerebellar Ataxia 3, run by National University of Malaysia at 1 site in Malaysia.
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Spinocerebellar Ataxia 3
- What is being tested?
- trehalose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 1 siteMalaysia
- Study dates
- 7 Mar 2018to 7 Sep 2018
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s)
- Consent to participate in the study
- The age of 18 years and older
Who cannot join
- Unconfirmed SCA 3
- Concomitant disorder(s) that affect SARA and other ataxia measures used in this study
- Diabetes
- Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of absorption results in diarrhoea and intestinal distress.
- Less than 18 years old
Trial locations and contacts
1 site in 1 country.
Malaysia1 site
Pusat Perubatan Universiti Kebangsaan Malaysia
Kuala Lumpur, 56000
About this study
Official title: Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study
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.
Read the detailed 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.
- Conditions
- Spinocerebellar Ataxia 3
- Being tested
- trehalose
- Medical subject headings
- Machado-Joseph Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
trehaloseDietary supplement
patients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily
| Study group | Role | What they receive |
|---|---|---|
| interventional | Experimental | supplement: trehalose Receives: Dietary Supplement: trehalose |
What is being measured
The primary outcome is the main question the study is designed to answer.
scale of rating of ataxia (SARA) score months,
Assessment of SARA scores by a single assessor
Measured: 2 monthly intervals for 6 months
SCA Functional Index Scores
Assessment of SCAFI by a single assessor
Measured: 2 monthly intervals for 6 months
EQ5D3L - quality of life scores
Assessment of quality of life scores
Measured: 2 monthly intervals for 6 months
Secondary outcomes (2)
Side effects Profile
Adverse Effects
Measured: 2 monthly intervals for 6 months
Blood investigation
Measurement of renal profile, fasting blood glucose, full blood count and liver profile
Measured: At baseline and at 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2020
- Study start7 Mar 2018
- Primary completion7 Sep 2018
- Study completion7 Sep 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- National University of Malaysia
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Dr Norlinah Mohamed Ibrahim, National University of Malaysia
- Investigators
- NORLINAH MOHAMED IBRAHIM, MBBCH, Principal investigator, norlinah@ppukm.ukm.edu.my
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04426149
- Sponsor's protocol ID
- UKM PPI/111/8/JEP-2017-826
- Status last verified
- Jun 2020
- First submitted
- 18 Apr 2019
- Last updated
- 11 Jun 2020
- Results posted
- No
- Data monitoring committee
- Yes
- 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 11 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3: A Pilot Study
- ClinicalTrials.gov ID
- NCT04426149
- Acronym / sponsor ID
- UKM PPI/111/8/JEP-2017-826
- Conditions
- Spinocerebellar Ataxia 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- National University of MalaysiaUniversity, hospital or other
- Study start
- 7 Mar 2018 (actual)
- Primary completion
- 7 Sep 2018 (actual)
- Study completion
- 7 Sep 2018 (actual)
- First posted
- 11 Jun 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.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| interventional | Experimental | Dietary Supplement: trehalosesupplement: trehalose |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- DNA diagnosis of SCA 3 in the study subject of his/ her affected family member(s)
- Consent to participate in the study
- The age of 18 years and older
Key exclusion criteria (5 listed)
- Unconfirmed SCA 3
- Concomitant disorder(s) that affect SARA and other ataxia measures used in this study
- Diabetes
- Malabsorption of trehalose underlies intolerance to mushrooms, since the lack of absorption results in diarrhoea and intestinal distress.
- Less than 18 years old
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 |
|---|---|---|
| trehalosepatients were instructed to ingest 100g of oral trehalose diluted in 500ml of water or other beverages daily | Dietary supplement | interventional |
Primary outcome
scale of rating of ataxia (SARA) score months,
Time frame: 2 monthly intervals for 6 months
Assessment of SARA scores by a single assessor
SCA Functional Index Scores
Time frame: 2 monthly intervals for 6 months
Assessment of SCAFI by a single assessor
EQ5D3L - quality of life scores
Time frame: 2 monthly intervals for 6 months
Assessment of quality of life scores
2 secondary outcomes
Side effects Profile
Time frame: 2 monthly intervals for 6 months
Blood investigation
Time frame: At baseline and at 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2020.
- 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, MBBCH principal investigator
norlinah@ppukm.ukm.edu.my
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 in people with Spinocerebellar Ataxia 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 3 inclusion and 5 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: finished, as of the sponsor's last update on 11 Jun 2020. 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. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
scale of rating of ataxia (SARA) score months, (time frame: 2 monthly intervals for 6 months). The study lists 3 primary outcomes in all. 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 11 Jun 2020.