Niemann-Pick disease type C
Conditions
Brief summary
The primary endpoint for the study is of the Scale for the Assessment and Rating of Ataxia (SARA). In the US, the Modified SARA is the primary endpoint.
Detailed description
Spinocerebellar Ataxia Functional Index (SCAFI), Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for patients aged <18 years, Modified Disability Rating Scale (mDRS), Physician’s, Caregiver’s (if applicable), and Patient’s (if able) Clinical Global Impression of Improvement (CGI-I) comparing end of period I (Visit 4) to baseline (Visit 2), and end of period II (Visit 6) to end of period I (Visit 4
Interventions
Sponsors
Intrabio Limited
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint for the study is of the Scale for the Assessment and Rating of Ataxia (SARA). In the US, the Modified SARA is the primary endpoint. | — |
Secondary
| Measure | Time frame |
|---|---|
| Spinocerebellar Ataxia Functional Index (SCAFI), Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for patients aged <18 years, Modified Disability Rating Scale (mDRS), Physician’s, Caregiver’s (if applicable), and Patient’s (if able) Clinical Global Impression of Improvement (CGI-I) comparing end of period I (Visit 4) to baseline (Visit 2), and end of period II (Visit 6) to end of period I (Visit 4 | — |
Countries
Czechia, Germany, Netherlands, Slovakia
Outcome results
None listed