Riluzole in Patients With Spinocerebellar Ataxia Type 7

NCT03660917 | Recruiting Phase 2

• 1 other identifier: AIFA-2016-02365063
• Study Type: interventional
• Target: 34
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

SCA7

Outcome Measures

Primary Outcomes (2)

• visual acuity expressed as log MAR units

  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

  18 months

• the proportion of patients with stable Scale for the assessment and rating of ataxia (SARA) score

  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.

  18 months

Secondary Outcomes (5)

• Farnsworth D15 Arrangement Test

  18 months

• Visual evoked potentials

  18 months

• Electroretinography

  18 months

• Optical Coherence tomography

  18 months

• Scale for the assessment and rating of ataxia (SARA) score

  18 months

Study Arms (2)

Riluzole

EXPERIMENTAL

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).

Drug: Riluzole

Placebo + riluzole

PLACEBO COMPARATOR

Placebo twice daily for 6 months and riluzole 50 mg twice daily for the following 6 months in the comparison group

Drug: Riluzole; Drug: Placebo

Interventions

Riluzole DRUG

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

Placebo + riluzole; Riluzole

Placebo DRUG

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.

Placebo + riluzole

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• positive genetic test for SCA7.

You may not qualify if:

• 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.

Sponsors & Collaborators

• S. Andrea Hospital: lead

Study Sites (1)

Neurological Unit, S. Andrea Hospital, Faculty of Medicine and Psychology, "Sapienza" University of Rome

Rome, 00139, Italy

RECRUITING

MeSH Terms

Interventions

Riluzole

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Central Study Contacts

Silvia Romano, MD, PhD

CONTACT

+390633776044; silvia.romano@uniroma1.it

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: We opted for a randomized, double-blind, placebo-controlled pilot trial with a lead-in phase. Moreover, the patients to be included in the placebo arm 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 Record Dates

• First Submitted: May 15, 2018
• First Posted: September 7, 2018
• Study Start: June 1, 2021
• Primary Completion: September 1, 2025
• Study Completion (Estimated): September 1, 2026
• Last Updated: December 2, 2024

Record last verified: 2024-11

Locations

IT (1)