Overview

Riluzole in Patients With Spinocerebellar Ataxia Type 7

Status:
Unknown status

Trial end date:
2021-01-01

Target enrollment:
0

Participant gender:
All

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.

Phase:

Phase 2/Phase 3

Accepts Healthy Volunteers?

Details

Lead Sponsor:

S. Andrea Hospital

Treatments:

Riluzole

Criteria

Inclusion Criteria:

- positive genetic test for SCA7.

Exclusion Criteria:

- cardiac arrhythmias;

- haematologic diseases;

- hepatic diseases with serum values of alanine aminotransferase, aspartate
aminotransferase or bilirubin > 1·5 times above normal limit;

- pregnancy (women of childbearing potential agreed to use contraception);

- breastfeeding.