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Trial of Sodium Valproate in Amyotrophic Lateral Sclerosis

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. Identifier: NCT00136110
Recruitment Status : Completed
First Posted : August 26, 2005
Last Update Posted : May 1, 2007
Princess Beatrix Muscle Foundation
Information provided by:
UMC Utrecht

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Sodium Valproate Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 165 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled Sequential Clinical Trial of Sodium Valproate in ALS
Study Start Date : April 2005
Actual Study Completion Date : February 2007

Primary Outcome Measures :
  1. Survival

Secondary Outcome Measures :
  1. The rate of decline of daily functioning

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Definite, probable, or probable-laboratory supported ALS according to the revised El Escorial World Federation of Neurology criteria.
  • Intake of riluzole 50 mg twice a day (bid)
  • A disease duration at inclusion of more than 6 months and less than 36 months [inclusive] (disease onset is defined as the date of first symptoms excluding muscle cramps and fasciculations)
  • Vital capacity (VC%) ≥ 70% of normal value (slow expiration, best of a minimum of three and a maximum of five measurements, with a respiratory function validly assessable and a spontaneous, non-assisted ventilation)
  • Ages 18 - 85 years (inclusive)
  • Capable of thoroughly understanding the trial information given; has signed the informed consent.

Exclusion Criteria:

  • Tracheostomy, tracheostomal ventilation of any type, non-invasive ventilation more than 16 hours/ day, or supplemental oxygen during the last three months prior to inclusion.
  • Any medical condition or intoxication known to have an association with motor neuron dysfunction, which might confound or obscure the diagnosis of ALS.
  • Presence of any concomitant life-threatening disease or any disease or impairment likely to interfere with functional assessment.
  • Confirmed hepatic insufficiency or abnormal liver function (ASAT, ALAT greater than twice the upper limit of normal range).

Information from the National Library of Medicine

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 identifier (NCT number): NCT00136110

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UMC Utrecht
Utrecht, Netherlands, 3584 CX
Sponsors and Collaborators
UMC Utrecht
Princess Beatrix Muscle Foundation
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Study Chair: Leonard H Van den Berg, MD, PhD UMC Utrecht
Principal Investigator: Sanne Piepers, MD UMC Utrecht
Principal Investigator: Sonja W De Jong, MD UMC Utrecht
Publications automatically indexed to this study by Identifier (NCT Number):
Layout table for additonal information Identifier: NCT00136110    
Other Study ID Numbers: 04/182-0
First Posted: August 26, 2005    Key Record Dates
Last Update Posted: May 1, 2007
Last Verified: April 2007
Keywords provided by UMC Utrecht:
Sodium Valproate
randomised trial
Amyotrophic Lateral Sclerosis (ALS)
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Valproic Acid
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Antimanic Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs