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Efficacy of Riluzole in Hereditary Cerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT01104649
Recruitment Status : Completed
First Posted : April 15, 2010
Last Update Posted : April 21, 2015
Sponsor:
Collaborator:
Agenzia Italiana del Farmaco
Information provided by (Responsible Party):
Giovanni Ristori, S. Andrea Hospital

April 7, 2010
April 15, 2010
April 21, 2015
April 2010
February 2014   (Final data collection date for primary outcome measure)
Scale for the assessment and rating of ataxia (SARA) [ Time Frame: 12 months ]
Improvement in ataxia
Same as current
Complete list of historical versions of study NCT01104649 on ClinicalTrials.gov Archive Site
  • Baropodometric parameters [ Time Frame: 12 months ]
  • Quality of life [ Time Frame: 12 months ]
    SF-36
  • Depression [ Time Frame: 12 months ]
    Beck Scale
  • Baropodometric parameters [ Time Frame: 12 months ]
  • Quality of life [ Time Frame: 12 months ]
    SF-36
  • Anxiety [ Time Frame: 12 months ]
    State Trait Anxiety Inventory
  • Depression [ Time Frame: 12 months ]
    Beck Scale
Not Provided
Not Provided
 
Efficacy of Riluzole in Hereditary Cerebellar Ataxia
Efficacy of Riluzole in Hereditary Cerebellar Ataxia: a Randomized Double-blind Placebo-controlled Trial.

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.

Not Provided
Interventional
Phase 2
Phase 3
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Cerebellar Ataxia
  • Drug: riluzole
    Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months.
    Other Name: Rilutek
  • Drug: Placebo comparator
    Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months.
    Other Name: Placebo
  • 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)
    Intervention: Drug: riluzole
  • 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)
    Intervention: Drug: Placebo comparator

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
60
Same as current
March 2014
February 2014   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patients with genetically confirmed diagnosis of hereditary cerebellar ataxia

Exclusion Criteria:

  • Concomitant experimental therapy for ataxia
  • Serious systemic illnesses
  • Pregnancy
Sexes Eligible for Study: All
14 Years to 70 Years   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
 
NCT01104649
FARM7KAJM7
Yes
Not Provided
Not Provided
Giovanni Ristori, S. Andrea Hospital
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
S. Andrea Hospital
April 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP