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Riluzole in Patients With Spinocerebellar Ataxia Type 7
Sponsor: S. Andrea Hospital
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.
Official title: Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in Phase
Key Details
Gender
All
Age Range
7 Years - Any
Study Type
INTERVENTIONAL
Enrollment
34
Start Date
2021-06-01
Completion Date
2026-09-01
Last Updated
2024-12-02
Healthy Volunteers
No
Conditions
Interventions
Riluzole
Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months in the treated group.
Placebo
Placebo drug for 6 months, however they will receive riluzole during the last 6 months of study, so that all patients will undergo the active drug in the last phase of the study.
Locations (1)
Neurological Unit, S. Andrea Hospital, Faculty of Medicine and Psychology, "Sapienza" University of Rome
Rome, Italy