Ignite Creation Date:
2024-05-05 @ 12:00 PM
Last Modification Date:
2024-10-26 @ 9:18 AM
Study NCT ID:
NCT00202397
Status:
COMPLETED
Last Update Posted:
2014-02-11
First Post:
2005-09-12
Brief Title:
Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia
Sponsor:
S Andrea Hospital
Organization:
S Andrea Hospital
Study Overview
Official Title:
Phase 2 Study of Riluzole Effects on Patients With Chronic Cerebellar Ataxia
Status:
COMPLETED
Status Verified Date:
2013-12
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:
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
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
Detailed Description:
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
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
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