Ataxia Telangiectasia (A-T)
Conditions
Brief summary
Safety endpoint : assessment of treatment-emergent adverse events (TEAEs), including serious AEs, discontinuations due to AEs, changes in vital signs, laboratory parameters, physical and neurological examination findings, and the C-SSRS. Additionally, potential effects associated with the use of eDSP will be assessed by changes in cortisol, glucose, cluster of differentiation CD4+ lymphocyte count, bone mineral density obtained using energy x-ray absorptiometry (DEXA), and growth and development
Detailed description
Exploratory Endpoint Assessments: Evaluation of the effects of eDSP will be assessed by the International Cooperative Ataxia Rating Scale (ICARS), the modified ICARS (mICARS), and the Rescored mICARS (RmICARS).
Sponsors
Quince Therapeutics S.p.A.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety endpoint : assessment of treatment-emergent adverse events (TEAEs), including serious AEs, discontinuations due to AEs, changes in vital signs, laboratory parameters, physical and neurological examination findings, and the C-SSRS. Additionally, potential effects associated with the use of eDSP will be assessed by changes in cortisol, glucose, cluster of differentiation CD4+ lymphocyte count, bone mineral density obtained using energy x-ray absorptiometry (DEXA), and growth and development | — |
Secondary
| Measure | Time frame |
|---|---|
| Exploratory Endpoint Assessments: Evaluation of the effects of eDSP will be assessed by the International Cooperative Ataxia Rating Scale (ICARS), the modified ICARS (mICARS), and the Rescored mICARS (RmICARS). | — |
Countries
Denmark, Germany, Italy, Norway, Poland, Spain
Outcome results
None listed