Huntington’s Disease
Conditions
Brief summary
Safety Endpoints: Safety profile as characterized by treatment-emergent adverse events (TEAEs), laboratory abnormalities, [CCI], electrocardiogram (ECG), vital signs, [CCI], [CCI], Columbia Suicide Severity Rating Scale (C SSRS), and physical examination, Primary Efficacy Endpoint: Change from Baseline in blood tHTT protein at Month 3
Detailed description
Change from Baseline in caudate volume as assessed via vMRI at Month 12 (key secondary), Change from Baseline in cUHDRS scores at Month 12, Change from Baseline in blood tHTT protein at Month 12, Change from Baseline in CSF mHTT protein at Month 12, Change from Baseline in blood mHTT protein at Month 12
Interventions
Sponsors
PTC Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety Endpoints: Safety profile as characterized by treatment-emergent adverse events (TEAEs), laboratory abnormalities, [CCI], electrocardiogram (ECG), vital signs, [CCI], [CCI], Columbia Suicide Severity Rating Scale (C SSRS), and physical examination, Primary Efficacy Endpoint: Change from Baseline in blood tHTT protein at Month 3 | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in caudate volume as assessed via vMRI at Month 12 (key secondary), Change from Baseline in cUHDRS scores at Month 12, Change from Baseline in blood tHTT protein at Month 12, Change from Baseline in CSF mHTT protein at Month 12, Change from Baseline in blood mHTT protein at Month 12 | — |
Countries
Austria, France, Germany, Italy, Netherlands, Spain
Outcome results
None listed