Spinocerebellar Ataxia type-3 (SCA3)
Conditions
Brief summary
Change from baseline in m-SARA total score at Week 52 in participants with SCA3 treated with SLS-005 0.75 g/kg compared to placebo
Detailed description
Key Secondary Endpoints In participants treated with SLS-005 0.75 g/kg: • Change from baseline in Clinical Global Impression of Severity (CGI-S) at Week 52, • Change from baseline in Patient Global Impression of Severity (PGI-S) at Week 52, • Change from baseline in Friedreich’s Ataxia Rating Scale – Activities of Daily Living (FARS-ADL) score at Week 52, • Change from baseline in m-SARA total score at Week 26, Secondary Endpoints In participants with treated with SLS-005 0.75 g/kg: • Changes from baseline in m-SARA total score at Weeks 4, 13, and 39, • Changes from baseline in CGI-S at Weeks 4, 13, 26, and 39, • Changes from baseline in PGI-S at Weeks 4, 13, 26, and 39, • Changes from baseline in FARS-ADL score at Weeks 4, 13, 26, and 39, • Changes from baseline in scores for each item of the m-SARA at Weeks 4, 13, 26, 39, and 52, In all participants: • Incidences of treatment-emergent adverse events (TEAEs) and SAEs, including clinically significant laboratory and ECG abnormalities
Sponsors
Eligibility
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in m-SARA total score at Week 52 in participants with SCA3 treated with SLS-005 0.75 g/kg compared to placebo | — |
Secondary
| Measure | Time frame |
|---|---|
| Key Secondary Endpoints In participants treated with SLS-005 0.75 g/kg: • Change from baseline in Clinical Global Impression of Severity (CGI-S) at Week 52, • Change from baseline in Patient Global Impression of Severity (PGI-S) at Week 52, • Change from baseline in Friedreich’s Ataxia Rating Scale – Activities of Daily Living (FARS-ADL) score at Week 52, • Change from baseline in m-SARA total score at Week 26, Secondary Endpoints In participants with treated with SLS-005 0.75 g/kg: • Changes from baseline in m-SARA total score at Weeks 4, 13, and 39, • Changes from baseline in CGI-S at Weeks 4, 13, 26, and 39, • Changes from baseline in PGI-S at Weeks 4, 13, 26, and 39, • Changes from baseline in FARS-ADL score at Weeks 4, 13, 26, and 39, • Changes from baseline in scores for each item of the m-SARA at Weeks 4, 13, 26, 39, and 52, In all participants: • Incidences of treatment-emergent adverse events (TEAEs) and SAEs, including clinically significant laboratory and ECG abnormalities | — |
Countries
Germany, Portugal, Spain