Ignite Creation Date:
2024-05-05 @ 11:48 AM
Last Modification Date:
2024-10-26 @ 9:13 AM
Study NCT ID:
NCT00136110
Status:
COMPLETED
Last Update Posted:
2007-05-01
First Post:
2005-08-24
Brief Title:
Trial of Sodium Valproate in Amyotrophic Lateral Sclerosis
Sponsor:
UMC Utrecht
Organization:
UMC Utrecht
Study Overview
Official Title:
A Randomized Double-Blind Placebo-Controlled Sequential Clinical Trial of Sodium Valproate in ALS
Status:
COMPLETED
Status Verified Date:
2007-04
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 purpose of this study is to determine whether the use of sodium valproate is effective in slowing the disease progression in Amyotrophic Lateral Sclerosis
Detailed Description:
Amyotrophic lateral sclerosis ALS is a devastating disease characterized by progressive degeneration of motor neurons leading to muscle weakness
The pathogenesis of ALS is unknown but there is convincing evidence that several molecular mechanisms play a role Previous studies investigated the role of the Survival Motor Neuron SMN gene in ALS Recent data suggest that SMN genotypes producing less SMN protein increase susceptibility and severity of ALS This leads to the hypothesis that the clinical expression of ALS is influenced by the total SMN protein level in affected patients In a population of ALS patients in the Netherlands we found that SMN genotypes producing less SMN protein appear to increase susceptibility and severity of ALS It was shown that the HDAC inhibitor sodium valproate SVP increases levels of SMN protein in vitro From these results and from data suggesting neuroprotective properties of SVP it is hypothesised that SVP could extend survival of patients with ALS In addition sodium valproate significantly prolonged the disease duration in the animal model for ALS the SOD1 transgenic mouse Given that SVP is a FDA-approved compound with well-known pharmacokinetic and toxicity profiles it is an attractive candidate for a clinical trial in ALS patients
The pathogenesis of ALS is unknown but there is convincing evidence that several molecular mechanisms play a role Previous studies investigated the role of the Survival Motor Neuron SMN gene in ALS Recent data suggest that SMN genotypes producing less SMN protein increase susceptibility and severity of ALS This leads to the hypothesis that the clinical expression of ALS is influenced by the total SMN protein level in affected patients In a population of ALS patients in the Netherlands we found that SMN genotypes producing less SMN protein appear to increase susceptibility and severity of ALS It was shown that the HDAC inhibitor sodium valproate SVP increases levels of SMN protein in vitro From these results and from data suggesting neuroprotective properties of SVP it is hypothesised that SVP could extend survival of patients with ALS In addition sodium valproate significantly prolonged the disease duration in the animal model for ALS the SOD1 transgenic mouse Given that SVP is a FDA-approved compound with well-known pharmacokinetic and toxicity profiles it is an attractive candidate for a clinical trial in ALS 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