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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

Tracking Information
First Submitted Date  ICMJE April 7, 2010
First Posted Date  ICMJE April 15, 2010
Last Update Posted Date April 21, 2015
Study Start Date  ICMJE April 2010
Actual Primary Completion Date February 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 14, 2010)
Scale for the assessment and rating of ataxia (SARA) [ Time Frame: 12 months ]
Improvement in ataxia
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01104649 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 20, 2015)
  • Baropodometric parameters [ Time Frame: 12 months ]
  • Quality of life [ Time Frame: 12 months ]
    SF-36
  • Depression [ Time Frame: 12 months ]
    Beck Scale
Original Secondary Outcome Measures  ICMJE
 (submitted: April 14, 2010)
  • 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy of Riluzole in Hereditary Cerebellar Ataxia
Official Title  ICMJE Efficacy of Riluzole in Hereditary Cerebellar Ataxia: a Randomized Double-blind Placebo-controlled Trial.
Brief Summary

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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Cerebellar Ataxia
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • 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
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 14, 2010)
60
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE March 2014
Actual Primary Completion Date February 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patients with genetically confirmed diagnosis of hereditary cerebellar ataxia

Exclusion Criteria:

  • Concomitant experimental therapy for ataxia
  • Serious systemic illnesses
  • Pregnancy
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 14 Years to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01104649
Other Study ID Numbers  ICMJE FARM7KAJM7
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Giovanni Ristori, S. Andrea Hospital
Study Sponsor  ICMJE S. Andrea Hospital
Collaborators  ICMJE Agenzia Italiana del Farmaco
Investigators  ICMJE
Principal Investigator: Silvia Romano, MD, PhD Center for Experimental Neurological Therapies (CENTERS), S. Andrea Hospital, II Faculty of Medicine, "Sapienza" University of Rome
PRS Account S. Andrea Hospital
Verification Date April 2015

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