Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia

• Status: Completed
• Trial end date: 2008-08-01
• Target enrollment: 0
• Participant gender: All

Summary

Cerebellar disorders are often disabling and symptomatic therapies are limited to few options that are partially effective. It seems therefore appropriate to search for additional approaches.

Purkinje cells are the sole output of the cerebellar cortex: they project inhibitory signals to the deep cerebellar nuclei (DCN), which have a critical role in cerebellar function and motor performance. DCN neurons fire spontaneously in the absence of synaptic input from Purkinje neurons and modulation of the DCN response by Purkinje input is believed to be responsible for coordination of movement. Recent evidences support the notion that an increase in DCN excitability may be an important step in the development of cerebellar ataxia and point to the underlying molecular mechanisms: the inhibition of small-conductance calcium-activated potassium (SK) channels, that causes an increase of the firing frequency in DCN, correlates with cerebellar ataxia.

The rationale of the present project is that SK channel openers, such as riluzole, may have a beneficial effect on cerebellar ataxia.

The researchers propose to perform a pilot study investigating safety and efficacy of riluzole, an approved treatment for amyotrophic lateral sclerosis, as a symptomatic approach in patients with chronic cerebellar ataxia.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: S. Andrea Hospital
• Treatments: Riluzole

Criteria

Inclusion Criteria:

• Patients with cerebellar degeneration (heredoataxias, sporadic idiopathic ataxia, multiple system atrophy type C)

• Patients who meet McDonald criteria for probable or definite multiple sclerosis (MS) with chronic cerebellar ataxia (not acute cerebellar ataxia due to relapse)

• Age between 18 and 80 years

Exclusion Criteria:

• Ataxia due to other diseases

• Acute cerebellar ataxia

• Use of other drugs for chronic ataxia

• Serious concomitant illnesses (cardiac arrhythmias, haematological and hepatic diseases)

• Pregnancy or breast feeding