A Phase 1b/2a first-in-human, multicentre, randomized, double-blind, placebo-controlled study of multiple ascending dose (Part1) followed by an open-label extension (Part2) to assess the safety, tolerability, and pharmacokinetics of intrathecally administered S233107 in participants with spinocerebellar ataxia type 3
Spinocerebellar ataxia type 3 (SCA3) is a rare inherited condition that affects movement, speech, and balance. Right now, there are no cures or treatments that can stop it from getting worse. This clinical trial is testing a new experimental medicine called S233107 to see if it is safe for people with SCA3. Because this is a early-stage study, researchers want to find out how the body tolerates different doses and how the drug moves through the bloodstream and spinal fluid. In the first part of the study, some people will receive the drug and others will receive a dummy injection (placebo). The medicine is given directly into the fluid around the spine. In the second part, all participants may receive the real medicine. Doctors will closely monitor participants' health, heart rhythms, and blood tests throughout.
At a glance
What is this study about?
Spinocerebellar ataxia type 3, sometimes called Machado-Joseph disease, is an inherited condition that causes gradual damage to parts of the nervous system. Over time, people with SCA3 may notice problems with their balance, walking, speech, and muscle control. Currently, treatments can only help manage everyday symptoms, but they cannot slow down or cure the underlying condition. Finding new medicines that directly target the disease is an urgent goal for doctors and families.
This clinical trial is looking at an experimental drug known as S233107. It is an early-stage study, meaning it is the very first time this drug has ever been tested in humans. Because of this, the main aim is not to prove that the drug cures the condition, but to check whether it is safe and tolerable. Researchers want to observe how well the body accepts the medicine and how it behaves inside the body.
The trial is split into two parts. In the first part, participants are given increasing doses, and some will receive a harmless dummy treatment called a placebo so doctors can compare the results fairly. In the second part, everyone who continues will receive the active study drug. The medicine is given by an injection into the lower back, directly into the fluid that surrounds the spinal cord. This allows the medicine to reach the nervous system where it is needed most.
Key takeaways
- Tests a new experimental drug called S233107 for spinocerebellar ataxia type 3.
- This is a first-in-human study focused primarily on safety and tolerability.
- The treatment is given through an injection into the spinal fluid.
- The study includes a comparison with a dummy treatment (placebo) before an open-label phase.
- Participants will have regular check-ups, including heart traces, blood tests, and spinal fluid checks.
Who may be eligible?
This trial is open to adults of any gender who are 18 years old or older and have a confirmed diagnosis of spinocerebellar ataxia type 3 (SCA3).
Because this is an early-phase trial testing a medicine in humans for the first time, there will be strict medical safety checks. People who have certain other serious health issues, heart problems, or medical reasons why they cannot safely have an injection into the spine may not be able to take part.
A doctor or research nurse at a trial clinic will go through your full medical history, run routine tests, and check your current medications to confirm whether taking part would be safe and appropriate for you.
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.
- I am at least 18 years old.
- I have been diagnosed with spinocerebellar ataxia type 3 (SCA3).
- I am able and willing to attend frequent hospital visits for tests and monitoring.
- I am comfortable receiving injections into the fluid around my spine.
What does participation involve?
If you take part, you will attend regular hospital appointments. The study drug is given as an injection into the fluid around your spinal cord, which is done through your lower back (a procedure similar to a lumbar puncture). In the first part of the study, you will be randomly chosen to receive either the active drug at a specific dose or a dummy injection (placebo). Neither you nor your study doctor will know which one you are receiving. Later in the study, all participants may receive the active medicine in an extension phase.
During your visits, the medical team will keep a close eye on your health. They will check your blood pressure and heart rate, perform heart traces (ECGs), and take regular blood and spinal fluid samples. They will also ask about your mood, mental wellbeing, and any side effects you notice. The study involves multiple clinic visits over several months to allow researchers to carefully track the drug's safety and how it breaks down in your body.
Potential risks and benefits
Locations (4)
- —UnverifiedFrance
- —UnverifiedDenmark
- —UnverifiedSpain
- —UnverifiedPortugal
Common questions
What is the main purpose of this study?
The main goal is to test the safety of the experimental drug S233107 and see how the human body handles it, rather than proving it is a cure.
How is the medicine given?
It is given as an injection into the spinal fluid in your lower back, similar to a routine lumbar puncture.
What is a placebo?
A placebo is a harmless dummy treatment that looks just like the real medicine but contains no active drug. It helps doctors make fair comparisons.
Will I definitely receive the real medicine?
In the first part of the study, you have a chance of receiving a placebo. In the second part, all participants who continue are scheduled to receive the active study medicine.
Can I leave the study if I change my mind?
Yes. Taking part is completely voluntary, and you can stop at any time without giving a reason and without affecting your normal healthcare.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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