Ongoing, recruitingPhase II and Phase III (Integrated)Interventional

A DOUBLE-BLIND, RANDOMIZED, PLACEBO CONTROLLED TRIAL TO ASSESS SAFETY AND EFFICACY OF SLS-005 (TREHALOSE INJECTION, 90.5 MG/ML FOR INTRAVENOUS INFUSION) FOR THE TREATMENT OF ADULTS WITH SPINOCEREBELLAR ATAXIA

This research is a study looking at a new medicine called SLS-005, which is given through a drip, for adults living with Spinocerebellar Ataxia type-3 (SCA3). The main goal is to find out if this medicine can help improve their movement and balance over a year, compared to a dummy medicine (placebo). Researchers will also check if it helps with daily tasks and how people feel about their condition. An important part of the study is also to make sure the medicine is safe and doesn't cause too many side effects. This study includes people of all genders, aged 18 and over, and will provide valuable information about a potential new treatment for SCA3.

At a glance

Status

Ongoing, recruiting

Phase

Phase II and Phase III (Integrated)

Sponsor

Seelos Therapeutics Inc.

Enrolment target

Start

13 Dec 2022

Spinocerebellar Ataxia type-3 (SCA3)

What is this study about?

This study is about a new potential medicine, called SLS-005, for people who have Spinocerebellar Ataxia type-3, often shortened to SCA3. SCA3 is a condition that affects your brain and spinal cord, causing problems with balance, coordination, and movement. Right now, there isn't a cure for SCA3, so doctors and researchers are always looking for new ways to help people manage their symptoms.

The main aim of this study is to see if SLS-005 can make a difference in how people with SCA3 move and coordinate themselves. They'll be looking at something called the 'm-SARA score', which is a way doctors measure how much SCA3 affects a person's balance, walking, speech, and other movements. The study will compare people who receive SLS-005 with those who receive a 'placebo' – which looks just like the real medicine but contains no active ingredient. This helps researchers understand if any improvements are truly due to the medicine or something else.

Besides looking at movement, the study will also check other important things. This includes how people feel about their condition and how well they can do everyday activities. Very importantly, the study wants to understand if SLS-005 is safe to use and what side effects it might cause. By gathering all this information, researchers hope to learn if SLS-005 could be a helpful and safe treatment option for adults with SCA3 in the future.

Key takeaways

This study is testing a new medicine (SLS-005) for adults with SCA3.
The main goal is to see if it improves movement and balance.
It's a 'double-blind' study, comparing the medicine to a placebo.
Safety and side effects will be carefully monitored.
Participation lasts for about one year with regular clinic visits.
Both men and women, aged 18 and over, can join.

Who may be eligible?

This study is open to adults who have been diagnosed with Spinocerebellar Ataxia type-3 (SCA3). You must be at least 18 years old to take part, and there's no upper age limit mentioned.

Both men and women are welcome to join this research study. The researchers are looking for a diverse group of participants to make sure the results apply widely to people with SCA3.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years of age or older?
Have you been diagnosed with Spinocerebellar Ataxia type-3 (SCA3)?
Are you able to attend regular clinic appointments for about a year?
Are you comfortable receiving medication through an intravenous drip?

Answer every question to see your result.

What does participation involve?

If you join this study, you would receive the study medicine, SLS-005, or a placebo (a dummy medicine). Both are given as an injection into a vein (like a drip) over a period of 52 weeks (about one year). You wouldn't know if you're getting the active medicine or the placebo, and neither would your study doctor, which helps to keep the study fair and accurate.

Throughout the year, you would have regular visits to the study clinic. During these visits, the research team would assess your movement and coordination using specific scales, ask you about your daily activities and how you feel, and conduct general health checks including blood tests and heart recordings (ECGs). These assessments would happen at several points, including weeks 4, 13, 26, 39, and 52 after starting the treatment, to track any changes. The total duration of active treatment and assessments in the study would be about one year.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. You might benefit from the new medicine if it proves to be effective in improving your symptoms. Even if you receive the placebo, you'll receive careful medical attention and monitoring throughout the study. However, SLS-005 is still being tested, so there's a chance it might not help, or it could cause side effects, some of which might be unpleasant or even serious. The research team will monitor you closely for any unwanted effects. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Spain
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Unverified
Portugal
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Unverified
Germany

Common questions

What is SCA3?

SCA3 is a condition that affects your brain and spinal cord, leading to problems with balance, coordination, and movement. It's also known as Machado-Joseph disease.

What is a 'placebo'?

A placebo is a 'dummy' medicine that looks exactly like the real study drug but contains no active ingredients. It helps researchers compare the real drug's effects accurately.

Will I know if I'm getting the real medicine or the placebo?

No, this is a 'double-blind' study, meaning neither you nor your study doctor will know who is receiving the active medicine and who is receiving the placebo. This helps ensure fair and unbiased results.

How is the medicine given?

The study medicine (SLS-005 or placebo) is given as an intravenous infusion, which means it's delivered directly into a vein through a drip.

How long will the study last for me?

If you take part, you would be involved in the study for about one year, including regular clinic visits and receiving the treatment.