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NCT Number: NCT00202397

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

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.

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Key information

Conditions

Hereditary Ataxia Ataxia Autoimmune Diseases Autoimmune Diseases of the Nervous System Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Immune System Diseases Multiple Sclerosis Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms Spinal Cord Diseases Spinocerebellar Degenerations

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

S.Andrea Hospital - University of Rome "La Sapienza"

Rome, 00100, Italy

About this study

Forty patients with chronic cerebellar ataxia will be enrolled in a double-bind, randomized, placebo-controlled trial.

By central randomisation, patients will take 50 mg of riluzole or placebo twice daily for 8 weeks.

Electrocardiogram routine laboratory tests and pregnancy tests will be performed before drug administration, after 4 weeks of treatment and at the end of the study (after 8 weeks of treatment).

At the same time points the International Cooperative Ataxia Rating Scale (ICARS) for pharmacological assessment of the cerebellar syndrome will be administered to the two groups (riluzole and placebo) of patients. To guarantee the evaluation of the results in blind conditions, the neurologists who will evaluate the ICARS scores will be different from those who will deal with randomisation and follow-up of patients.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Riluzole

Drug

capsule-shaped 50 mg tablets bid for 8 weeks

Other names: Rilutek ATC Code N07X X02

Placebo

Other

capsule-shaped tablet bid for 8 weeks

Primary outcomes

  1. The International Cooperative Ataxia Rating Scale (ICARS) total scores and subscores (oculomotor, kinetic, postural, speech), comparing the three time points in the treated versus placebo group

    Time frame: pre-treatment, after 4 weeks of treatment and at the end of the study

Sponsors and collaborators

Lead sponsor

S. Andrea Hospital

Other

Registry information

Official study title

Phase 2 Study of Riluzole Effects on Patients With Chronic Cerebellar Ataxia

Important dates

Study start
2005
Primary completion
2008
Study completion
2008
First posted
Sep 20, 2005
Registry last updated
Dec 2, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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