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.
Study 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
Sex
ALL
Min age
7 Years
Max age
—
Healthy volunteers
No
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.
Primary outcome measure(s)
visual acuity expressed as log MAR units — 18 months the metric to quantify the best corrected visual acuity, by applying the ETDRS chart (either back-illuminated or projected) with the patient's correction for distance
the proportion of patients with stable Scale for the assessment and rating of ataxia (SARA) score — 18 months neurological assessment for ataxia. It has eight categories with accumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia). When completing the outcome measure each category is assessed and scored accordingly. Scores for the eight items range as follows:
Gait (0-8 points), Stance (0-6 points), Sitting (0-4 points) Speech disturbance (0-6 points) Finger chase (0-4 points) Nose-finger test (0-4 points) Fast alternating hand movement (0-4 points) Heel-shin slide (0-4 points) Once each of the 8 categories have been assessed, the total is calculated to determine the severity of ataxia.
For motor activities of the four extremities (items 5-8), assessments are performed bilaterally, and the mean values are used to obtain the total score.
Trial sites (1)
Facility
City
Region
Status
Neurological Unit, S. Andrea Hospital, Faculty of Medicine and Psychology, "Sapienza" University of Rome
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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