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Efficacy of Riluzole in Hereditary Cerebellar Ataxia

This study has been completed.
Agenzia Italiana del Farmaco
Information provided by (Responsible Party):
Giovanni Ristori, S. Andrea Hospital Identifier:
First received: April 7, 2010
Last updated: April 20, 2015
Last verified: April 2015

The hereditary cerebellar ataxias include diverse neurodegenerative disorders. Hereditary ataxias can be divided into autosomal dominant ataxias (ADCAs), autosomal recessive ataxias (ARCAs), X-linked, and mitochondrial ataxias on the basis of mode of inheritance. The key feature in all these disorders is ataxia typically characterised by poor balance, hand incoordination, postural or kinetic tremor, dysarthria and dysphagia.

To date no treatment has been shown to slow progression of the disease and symptomatic therapies are limited to few options that are partially effective.

Purkinje cells 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, while uncontrolled spontaneous firing of DCN neurons may underlay cerebellar ataxia. Recent studies have demonstrated that small-conductance calcium-activated potassium (SK) channels inhibitor are able to increase DCN firing rate. Since SK channels are critical regulators of DCN firing rate, SK openers such as the drug riluzole may reduce neuronal hyperexcitability and thereby be useful in the therapy of cerebellar ataxia.

On this base the investigators published a pilot study in patients with chronic cerebellar ataxia (Ristori et al., Neurology 2010) investigating safety and efficacy of riluzole or placebo administration for 8 weeks. The results demonstrated a significative improvement in International Cooperative Ataxia Rating Scale (ICARS) global score after four weeks and after 8 weeks in the riluzole arm.

The present protocol is aimed at verifying the safety and efficacy of riluzole administration for a longer period, in a larger sample size of patients, with more stringent diagnostic criteria (hereditary cerebellar ataxia), respect to the above pilot study. Sixty patients will be enrolled in a double-blind, placebo-controlled trial. By central randomisation, patients will take 50 mg of riluzole or placebo twice daily for 12 months. Treatment effects will be assessed by comparing the Scale for the Assessment and Rating of Ataxia (SARA) before treatment and during therapy at months 3 and 12.

Condition Intervention Phase
Cerebellar Ataxia
Drug: riluzole
Drug: Placebo comparator
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Efficacy of Riluzole in Hereditary Cerebellar Ataxia: a Randomized Double-blind Placebo-controlled Trial.

Resource links provided by NLM:

Further study details as provided by S. Andrea Hospital:

Primary Outcome Measures:
  • Scale for the assessment and rating of ataxia (SARA) [ Time Frame: 12 months ]
    Improvement in ataxia

Secondary Outcome Measures:
  • Baropodometric parameters [ Time Frame: 12 months ]
  • Quality of life [ Time Frame: 12 months ]

  • Depression [ Time Frame: 12 months ]
    Beck Scale

Enrollment: 60
Study Start Date: April 2010
Study Completion Date: March 2014
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Riluzole
Riluzole 50 mg is administered orally every 12 hours for 12 months (a 2:1 ratio for SCA/FA in the stratified randomization procedure)
Drug: riluzole
Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months.
Other Name: Rilutek
Placebo Comparator: Placebo comparator
Placebo is administered orally every 12 hours for 12 months (a 2:1 ratio for SCA/FA in the stratified randomization procedure)
Drug: Placebo comparator
Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months.
Other Name: Placebo


Ages Eligible for Study:   14 Years to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients with genetically confirmed diagnosis of hereditary cerebellar ataxia

Exclusion Criteria:

  • Concomitant experimental therapy for ataxia
  • Serious systemic illnesses
  • Pregnancy
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Please refer to this study by its identifier: NCT01104649

Center for Experimental Neurological Therapies (CENTERS), S. Andrea Hospital, II Faculty of Medicine, "Sapienza" University of Rome
Rome, Italy, 00139
Sponsors and Collaborators
S. Andrea Hospital
Agenzia Italiana del Farmaco
Principal Investigator: Silvia Romano, MD, PhD Center for Experimental Neurological Therapies (CENTERS), S. Andrea Hospital, II Faculty of Medicine, "Sapienza" University of Rome
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Giovanni Ristori, MD, PhD, S. Andrea Hospital Identifier: NCT01104649     History of Changes
Other Study ID Numbers: FARM7KAJM7
Study First Received: April 7, 2010
Last Updated: April 20, 2015

Keywords provided by S. Andrea Hospital:
Spinocerebellar ataxia
Friedreich ataxia

Additional relevant MeSH terms:
Cerebellar Ataxia
Spinocerebellar Degenerations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents processed this record on May 24, 2017