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

This study has been completed.
Sponsor:
Information provided by:
S. Andrea Hospital
ClinicalTrials.gov Identifier:
NCT00202397
First received: September 12, 2005
Last updated: February 10, 2014
Last verified: December 2013

September 12, 2005
February 10, 2014
June 2005
June 2008   (final data collection date for primary outcome measure)
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 ] [ Designated as safety issue: No ]
  • The ICARS total scores, comparing the three time points (pre-treatment, after 4 weeks of treatment and at the end of the study) and the treated vs placebo group
  • The ICARS subscores (oculomotor, kinetic, postural, speech) comparing the three time points (pre-treatment, after 4 weeks of treatment and at the end of the study) and the treated vs placebo group
Complete list of historical versions of study NCT00202397 on ClinicalTrials.gov Archive Site
Not Provided
No secondary outcomes
Not Provided
Not Provided
 
Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia
Phase 2 Study of Riluzole Effects on 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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
  • Hereditary Ataxia
  • Multiple Sclerosis
  • Cerebellar Ataxia
  • Drug: Riluzole
    capsule-shaped 50 mg tablets bid for 8 weeks
    Other Name: Rilutek ATC Code N07X X02
  • Other: placebo
    capsule-shaped tablet bid for 8 weeks
  • Placebo Comparator: 2
    placebo bid for 8 weeks
    Intervention: Other: placebo
  • Experimental: 1
    Riluzole, capsule-shaped 50 mg tablets bid for 8 weeks
    Intervention: Drug: Riluzole

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
40
August 2008
June 2008   (final data collection date for primary outcome measure)

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
Both
18 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
NCT00202397
NEU - RLZ - 05
Yes
Giovanni Ristori, S.Andrea Hospital - II Faculty of Medicine, "Sapienza" University of Rome
S. Andrea Hospital
Not Provided
Study Director: Marco Salvetti, Assoc. Prof S.Andrea Hospital, University of Rome "La Sapienza"
Principal Investigator: Giovanni Ristori, MD University of Roma La Sapienza
S. Andrea Hospital
December 2013

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