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
Condition studied
Spinocerebellar ataxia type 3
Investigational medicinal product(s)
Eligibility
Primary endpoint
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.
Endpoint detail
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.
This page summarises publicly available CTIS data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View 2025-525111-17-00 on CTIS ↗ ← All trials in the EU