Health TrialsbyHealthPanel

Riluzole in Patients With Spinocerebellar Ataxia Type 7

ConditionSCA7
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age7+
SponsorS. Andrea Hospital

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

positive genetic test for SCA7.

Disqualifiers

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);

Trial design

Design model

Parallel

Treatments tested in this trial

  • Riluzole

    Drug

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

  • 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.

Treatment groups

34 Participants
are divided into 2 treatment groups
Group A: RiluzoleExperimental treatment 1 intervention
Group B: Placebo + riluzolePlacebo comparator 2 interventions

Trial outcomes

Primary outcomes

1

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

Time frame
18 months
2

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.

Time frame
18 months

Secondary outcomes

1

Farnsworth D15 Arrangement Test

quantitative ophthalmologic assessments

Time frame
18 months
2

Visual evoked potentials

quantitative ophthalmologic assessments

Time frame
18 months
3

Electroretinography

quantitative ophthalmologic assessments

Time frame
18 months
4

Optical Coherence tomography

quantitative ophthalmologic assessments

Time frame
18 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

S. Andrea Hospital

Lead sponsor

Source ClinicalTrials.gov
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