Spinocerebellar ataxia type 7 (SCA7)
Conditions
Brief summary
The primary endpoints will be the proportion of patients with stable SARA score and visual acuity expressed as log MAR units at 18 months, in comparison with the same parameters as mean of t0-t3-t6 evaluations .
Detailed description
The secondary endpoint will be quantitative ophthalmologic assessments (via a Farnsworth D15 Arrangement Test, Visual evoked, Electroretinography, Optical Coherence tomography, Computerized visual field examination) and SARA score as continuous values at 18 months, in comparison with the same parameters calculated for each patient as mean of t0-t3-t6 evaluations, The safety profile will be assessed through the recording, reporting and analyzing of baseline medical conditions, adverse events, physical examination findings including laboratory tests.
Interventions
DRUGGlentek 50 mg filmtabletten
DRUGPlacebo Riluzole is a hard gelatin capsule filled with the inert excipient microcrystalline cellulose (Avicel® PH200).
Sponsors
Universita Degli Studi Di Roma La Sapienza
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoints will be the proportion of patients with stable SARA score and visual acuity expressed as log MAR units at 18 months, in comparison with the same parameters as mean of t0-t3-t6 evaluations . | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary endpoint will be quantitative ophthalmologic assessments (via a Farnsworth D15 Arrangement Test, Visual evoked, Electroretinography, Optical Coherence tomography, Computerized visual field examination) and SARA score as continuous values at 18 months, in comparison with the same parameters calculated for each patient as mean of t0-t3-t6 evaluations, The safety profile will be assessed through the recording, reporting and analyzing of baseline medical conditions, adverse events, physical examination findings including laboratory tests. | — |
Countries
Italy
Outcome results
None listed