Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00202397|
Recruitment Status : Completed
First Posted : September 20, 2005
Last Update Posted : February 11, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Ataxia Multiple Sclerosis Cerebellar Ataxia||Drug: Riluzole Other: placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Triple (Participant, Investigator, Outcomes Assessor)|
|Official Title:||Phase 2 Study of Riluzole Effects on Patients With Chronic Cerebellar Ataxia|
|Study Start Date :||June 2005|
|Primary Completion Date :||June 2008|
|Study Completion Date :||August 2008|
Placebo Comparator: 2
placebo bid for 8 weeks
capsule-shaped tablet bid for 8 weeks
Riluzole, capsule-shaped 50 mg tablets bid for 8 weeks
capsule-shaped 50 mg tablets bid for 8 weeks
Other Name: Rilutek ATC Code N07X X02
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00202397
|S.Andrea Hospital - University of Rome "La Sapienza"|
|Rome, Italy, 00100|
|Study Director:||Marco Salvetti, Assoc. Prof||S.Andrea Hospital, University of Rome "La Sapienza"|
|Principal Investigator:||Giovanni Ristori, MD||University of Roma La Sapienza|