Study on Trehalose Dihydrate for Treating Spinocerebellar Ataxia in Adults

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What is this study about?

This clinical trial is focused on studying a condition called Spinocerebellar Ataxia type-3 (SCA3), which affects the brain and can lead to problems with movement and coordination. The study is testing a new treatment called SLS-005, which is a solution given through an intravenous infusion. The main ingredient in this treatment is trehalose dihydrate, a type of sugar that is being investigated for its potential benefits in treating SCA3.

The purpose of the study is to determine how effective SLS-005 is in treating adults with SCA3. Participants in the study will receive either the SLS-005 treatment or a placebo, which looks like the treatment but does not contain the active ingredient. The study will last for about a year, during which participants will receive regular infusions and attend follow-up visits to monitor their health and any changes in their condition.

Throughout the study, researchers will assess various aspects of the participants’ health, including their ability to perform daily activities and any changes in their symptoms. The goal is to see if SLS-005 can improve the quality of life for people with SCA3 by reducing the severity of their symptoms. Participants will be closely monitored for any side effects or adverse reactions to ensure their safety during the trial.

1 joining the study

Upon joining the study, you will be asked to provide a signed informed consent. This is a document that confirms your understanding of the study and your willingness to participate.

You will undergo a screening process to confirm your eligibility. This includes verifying your age, medical condition, and other criteria such as your Body Mass Index (BMI) and medication stability.

2 initial assessment

An initial assessment will be conducted to establish a baseline for your condition. This involves measuring your m-SARA score, which evaluates your symptoms related to spinocerebellar ataxia.

Additional assessments may include the Clinical Global Impression of Severity (CGI-S) and the Patient Global Impression of Severity (PGI-S).

3 treatment phase

You will receive the study medication, SLS-005, or a placebo through an intravenous infusion. The dosage is 0.75 grams per kilogram of your body weight.

The treatment will be administered regularly over a period of 52 weeks. The frequency of administration will be explained to you by the study team.

4 ongoing assessments

Throughout the study, your condition will be monitored at various intervals. Key assessments will occur at weeks 4, 13, 26, 39, and 52.

These assessments will include changes in your m-SARA score, CGI-S, PGI-S, and Friedreich’s Ataxia Rating Scale – Activities of Daily Living (FARS-ADL) score.

5 safety monitoring

Your safety will be closely monitored for any treatment-emergent adverse events (TEAEs) or serious adverse events (SAEs).

Regular laboratory tests and ECGs (electrocardiograms) will be conducted to ensure your well-being.

6 completion of the study

At the end of the 52-week period, a final assessment will be conducted to evaluate the overall impact of the treatment.

You will be informed about the results of the study and any potential next steps.

Who Can Join the Study?

Signed informed consent, which means you agree to participate after understanding the study.
Men and women aged between 18 and 75 years old.
A clinical diagnosis of Spinocerebellar Ataxia type-3 (SCA3) confirmed by genetic testing.
A total score of 4 or higher on the m-SARA test at the screening visit. The m-SARA is a scale used to measure the severity of ataxia symptoms.
A score of 1 or higher on the gait component of the m-SARA test at the screening visit. This part of the test measures walking ability.
Body Mass Index (BMI) between 18 and 35. BMI is a measure of body fat based on height and weight.
Stable doses of all other medications for at least 30 days before the screening visit.
A negative pregnancy test result at the screening visit for female participants who can become pregnant.
Willingness to follow the study’s guidelines on sexual abstinence or use of contraception.

Who Cannot Join the Study?

Patients who do not have a confirmed diagnosis of Spinocerebellar Ataxia type-3 (SCA3) cannot participate.
Patients who are not adults are excluded from the study.
Patients who are unable to provide informed consent are not eligible.
Patients with other serious health conditions that might interfere with the study are excluded.
Pregnant or breastfeeding women cannot participate.
Patients who are currently participating in another clinical trial are not eligible.
Patients with a history of allergic reactions to the study medication are excluded.
Patients who have used certain medications that might interfere with the study within a specific time frame before the study starts are not eligible.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Universitaet Leipzig	Leipzig	Germany
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Fundacio De Recerca Clinic Barcelona-Institut D’Investigacions Biomediques August Pi I Sunyer	Barcelona	Spain
Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.	Bonn	Germany
Cwmrfs Hnjpijmszw Ubqcpxhkojgqr Dv Pjmlx Enughr	Porto	Portugal

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	01.12.2022
Portugal	Not recruiting	01.12.2022
Spain	Not recruiting	01.12.2022

Trial locations

Investigated drugs:

Trehalose Dihydrate (Ph.eur.)

SLS-005 (Trehalose Injection) is a treatment being tested for its ability to help people with a condition called spinocerebellar ataxia type-3, which affects movement and coordination. This medication is given through an intravenous infusion, meaning it is delivered directly into the bloodstream through a vein. The goal of using SLS-005 is to see if it can improve symptoms or slow down the progression of this condition in adults.

Spinocerebellar Ataxia type-3 (SCA3) – Spinocerebellar Ataxia type-3, also known as Machado-Joseph disease, is a genetic disorder that affects the central nervous system. It is characterized by progressive problems with movement, including unsteady gait and lack of coordination. Over time, individuals may experience muscle stiffness, weakness, and involuntary eye movements. The disease can also lead to difficulties with speech and swallowing. As it progresses, it may affect other systems, causing symptoms such as sleep disturbances and neuropathy. The progression and severity of symptoms can vary widely among individuals.

Trial ID:

2022-501004-10-00

Protocol code:

SLS-005-302

Trial Phase:

Therapeutic use (Phase IV)

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Study on Trehalose Dihydrate for Treating Spinocerebellar Ataxia in Adults

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