Ignite Creation Date:
2024-05-05 @ 10:26 PM
Last Modification Date:
2024-10-26 @ 10:18 AM
Study NCT ID:
NCT01104649
Status:
COMPLETED
Last Update Posted:
2015-04-21
First Post:
2010-04-07
Brief Title:
Efficacy of Riluzole in Hereditary Cerebellar Ataxia
Sponsor:
S Andrea Hospital
Organization:
S Andrea Hospital
Study Overview
Official Title:
Efficacy of Riluzole in Hereditary Cerebellar Ataxia a Randomized Double-blind Placebo-controlled Trial
Status:
COMPLETED
Status Verified Date:
2015-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 nucleiDCN 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
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 nucleiDCN 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:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None