CompletedNot Applicable

Clinical Effects of Oral Trehalose In Patients With Spinocerebellar Ataxia 3

Malaysia13 participantsStarted 2018-03-07

Plain-language 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. 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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

Exclusion criteria

. 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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

scale of rating of ataxia (SARA) score months,

Timeframe: 2 monthly intervals for 6 months

SCA Functional Index Scores

Timeframe: 2 monthly intervals for 6 months

EQ5D3L - quality of life scores

Timeframe: 2 monthly intervals for 6 months

Trial details

NCT IDNCT04426149

SponsorNational University of Malaysia

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2018-09-07

View on ClinicalTrials.gov More Spinocerebellar Ataxia 3 trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. 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

Exclusion criteria

. 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.