CACNA1A Disorders
Conditions
Brief summary
The primary endpoint measure is the Scale for the Assessment and Rating of Ataxia (SARA). SARA is an eight-item clinical rating scale (range 0–40, where 0 is the best neurological status and 40 the worst). It is a reliable and valid clinical scale with a high internal consistency that measures the severity of ataxia and increases with ataxia disease stage.
Detailed description
• Spinocerebellar Ataxia Functional Index (SCAFI) • Functional Scale for the Assessment and Rating of Ataxia (f-SARA) [US only - key secondary endpoint in the US] • Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for children aged <18 years • Neuro Quality of Life – Upper Extremity Function (NeuroQOL-UEF) assessed by the Patient or the Caregiver (if patient unable to complete)
Interventions
Sponsors
Intrabio Limited, IntraBio Inc
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint measure is the Scale for the Assessment and Rating of Ataxia (SARA). SARA is an eight-item clinical rating scale (range 0–40, where 0 is the best neurological status and 40 the worst). It is a reliable and valid clinical scale with a high internal consistency that measures the severity of ataxia and increases with ataxia disease stage. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Spinocerebellar Ataxia Functional Index (SCAFI) • Functional Scale for the Assessment and Rating of Ataxia (f-SARA) [US only - key secondary endpoint in the US] • Quality of Life EQ-5D-5L for patients aged ≥18; EQ-5D-Y for children aged <18 years • Neuro Quality of Life – Upper Extremity Function (NeuroQOL-UEF) assessed by the Patient or the Caregiver (if patient unable to complete) | — |
Outcome results
None listed