Ataxia Telangiectasia
Conditions
Brief summary
Primary Efficacy Endpoint= To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 compared to placebo in A-T (6- to 9-year-old participants primary analysis population).
Detailed description
Key Secondary Efficacy Endpoint = To evaluate the overall clinical effect of EryDex, compared to placebo, in A-T (6- to 9-year-old participants primary analysis population), based on CGI-S from baseline to Visit 9 (Day 168), Other Secondary Efficacy Endpoint = CGI-C from baseline to Visit 9 (Day 168)
Interventions
DRUGSodium chloride 0.372% solution
Sponsors
Quince Therapeutics S.p.A.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary Efficacy Endpoint= To evaluate the effect of EryDex on CNS symptoms, as measured by the change of the RmICARS from baseline to Visit 9 compared to placebo in A-T (6- to 9-year-old participants primary analysis population). | — |
Secondary
| Measure | Time frame |
|---|---|
| Key Secondary Efficacy Endpoint = To evaluate the overall clinical effect of EryDex, compared to placebo, in A-T (6- to 9-year-old participants primary analysis population), based on CGI-S from baseline to Visit 9 (Day 168), Other Secondary Efficacy Endpoint = CGI-C from baseline to Visit 9 (Day 168) | — |
Countries
Belgium, Denmark, Germany, Italy, Norway, Poland, Spain
Outcome results
None listed