In short
S233107 is an investigational substance being studied for spinocerebellar ataxia type 3 (SCA3), a rare inherited neurological condition. The clinical research programme includes one completed Phase 1/2 trial that enrolled 62 participants and tested the safety and tolerability of multiple intrathecal doses of S233107 compared with placebo. The study was a first-in-human, multicentre, randomised, double-blind, placebo-controlled multiple ascending dose trial with an open-label extension.
Key points
- S233107 is an investigational substance given by intrathecal injection, studied in spinocerebellar ataxia type 3.
- The single registered trial is a Phase 1/2 first-in-human study with 62 participants.
- The trial design was randomised, double-blind and placebo-controlled, followed by an open-label extension.
- The primary focus was safety and tolerability of multiple ascending intrathecal doses.
- The trial status is completed.
What is S233107?
S233107 is an investigational substance that has not been authorised as a medicine. The available registry records describe it only as a drug product given by intrathecal injection, meaning it is injected into the fluid surrounding the spinal cord. The substance is being studied in people with spinocerebellar ataxia type 3 (SCA3), a rare inherited condition that affects coordination and balance.
How does S233107 work?
The registry data and supplementary sources do not describe the molecular target or mechanism of action of S233107. The trial record states only that the substance is administered intrathecally as a solution for injection. Because no source explains how S233107 acts in the body, this section cannot be filled with specific information.
Which conditions is it used or studied for?
- Spinocerebellar ataxia type 3 (SCA3) – also known as Machado-Joseph disease, this is a rare inherited disorder that causes progressive problems with coordination, balance, speech and eye movements. It is the only condition listed in the registered trial for S233107.
How is S233107 given?
In the registered trial, S233107 was given as a solution for injection by the intrathecal route, meaning it was injected directly into the cerebrospinal fluid around the spinal cord. Two strengths were used: 15 mg/mL and 4 mg/mL. The trial involved multiple intrathecal administrations.
What do the clinical trials show?
One clinical trial of S233107 is registered. It is a Phase 1/2, first-in-human, multicentre, randomised, double-blind, placebo-controlled study that enrolled 62 participants with spinocerebellar ataxia type 3. The study had two parts: Part 1 compared multiple ascending intrathecal doses of S233107 with placebo, and Part 2 was an open-label extension in which all participants received S233107. The trial is recorded as completed.
The primary outcomes measured were the incidence and severity of adverse events, and the incidence of abnormalities in 12-lead electrocardiogram (ECG) readings, clinical laboratory assessments in blood and cerebrospinal fluid (CSF), vital signs, weight, and suicide risk. The trial did not list efficacy endpoints such as changes in ataxia symptoms.
The trial enrolled adults with spinocerebellar ataxia type 3. The registry record does not provide further detail on specific inclusion or exclusion criteria. The study was a first-in-human trial, meaning it was the first time S233107 was given to people.
What side effects are known?
The registered trial measured the incidence and severity of adverse events as its primary outcome, but the registry record does not report the results of that measurement. The supplementary source also does not provide a list of side effects. Therefore, no specific side effects of S233107 can be described from the available data.