drugset / Trial / NCT03660917

Riluzole in Patients With Spinocerebellar Ataxia Type 7

NCT03660917

Phase 2/3 Recruiting 34 enrolled S. Andrea Hospital
RandomizedParallel-groupDouble-blindTreatment

Summary

Spinocerebellar ataxia type 7 (SCA7) belongs to the dominant forms of inherited cerebellar ataxias (CA), being one of the rarest form. SCA7 has no therapeutic options, so that the relentless course, the important visual deficit that accompanies CA, and the possibility of disease development in childhood are pressing unmet needs. The investigators published encouraging data on riluzole in inherited CA other than SCA7. These results prompted off-label use of riluzole in single cases of SCA7 in Italy and United States, suggesting possible efficacy of the drug in this condition.

Timeline

Start
2021-06-01
Primary completion
2025-09-01
Completion
2026-09-01

Drugs

EvaluationDrugModalityDoseRoute
Subject Riluzole Small molecule 50 mg Oral