RecruitingPhase 2Phase 3NCT03660917

Riluzole in Patients With Spinocerebellar Ataxia Type 7

Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in Phase

Sponsor

S. Andrea Hospital

Enrollment

34 participants

Start Date

Jun 1, 2021

Study Type

INTERVENTIONAL

Conditions

SCA7

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

Min Age: 7 Years

Inclusion Criteria1

positive genetic test for SCA7.

Exclusion Criteria5

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.

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Interventions

DRUGRiluzole

Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months in the treated group.

DRUGPlacebo

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

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NCT03660917