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

Status

Not yet recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2025-525111-17-00

Enrollment

Registered

2026-07-30

Start date

Unknown

Completion date

Unknown

Last updated

2026-07-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinocerebellar ataxia type 3

Brief summary

Part 1 and 2: - Incidence and severity of AEs - Incidence of abnormalities in 12-lead ECG, clinical laboratory assessments in blood and CSF, vital signs, weight, and suicide risk.

Detailed description

Part 1 and 2: - Concentrations and derived PK parameters of S233107 in CSF and plasma, including but not limited to CSF Ctrough, plasma Cmax and plasma AUC0-tau.

Interventions

DRUGS233107 Solution for injection 15 mg/mL

DRUGS233107 Solution for injection 4 mg/mL

DRUGPlacebo matching S233107

Eligibility

Sex/Gender

All

Age

18 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Part 1 and 2: - Incidence and severity of AEs - Incidence of abnormalities in 12-lead ECG, clinical laboratory assessments in blood and CSF, vital signs, weight, and suicide risk.	—

Secondary

Measure	Time frame
Part 1 and 2: - Concentrations and derived PK parameters of S233107 in CSF and plasma, including but not limited to CSF Ctrough, plasma Cmax and plasma AUC0-tau.	—

Outcome results

None listed

Source: EU CTIS · Data processed: Aug 1, 2026