Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in Phase
Who is this study for? Patients with spinocerebellar ataxia type 7
What treatments are being studied? Riluzole
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 7
Healthy Volunteers: f
View:
• positive genetic test for SCA7.
Locations
Other Locations
Italy
Neurological Unit, S. Andrea Hospital, Faculty of Medicine and Psychology, Sapienza University of Rome
RECRUITING
Rome
Contact Information
Primary
Silvia Romano, MD, PhD
silvia.romano@uniroma1.it
+390633776044
Time Frame
Start Date: 2021-06-01
Estimated Completion Date: 2026-09-01
Participants
Target number of participants: 34
Treatments
Experimental: Riluzole
Riluzole 50 mg twice daily for 12 months in the treated group. In pre-pubertal subjects the dosage will be adjusted on a mg/m2 basis according to the recommended human daily dose (RHDD; 100 mg).
Placebo_comparator: Placebo + riluzole
Placebo twice daily for 6 months and riluzole 50 mg twice daily for the following 6 months in the comparison group
Related Therapeutic Areas
Sponsors
Leads: S. Andrea Hospital