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Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid

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ClinicalTrials.gov Identifier: NCT01033331
Recruitment Status : Completed
First Posted : December 16, 2009
Last Update Posted : December 16, 2009
Information provided by:

Study Description
Brief Summary:
The purpose of this study is to determine if the treatment with valproic acid can increase the muscle strength and motor ability of children with spinal muscular atrophy.

Condition or disease
Spinal Muscular Atrophy

Detailed Description:
Spinal muscular atrophy (SMA) is an autosomal recessive disorder that affects the motoneurons of the spinal anterior corn, resulting in hypotonia and muscle weakness. The knowledge about its molecular mechanism has led to clinical tests with drugs that increase survival motor neuron (SMN) protein level. The valproic acid (VA) that acts as a histone deacetylase inhibitor activates the SMN2 gene increasing the protein level. Methods: Twenty-two patients with type II and III SMA, aged between 2 and 18 years old, were treated with VA and were evaluated five times along a period of one year using the Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score, and the Barthel Index. The first evaluation was coincident with the introduction of VA. Results: After 12 months of therapy, the patients did not gain muscle strength but their motor ability has improved. Children younger than 6 years of age had a higher pronounced gain in motor ability. Conclusion: treatment of SMA patients with VA is one alternative to alleviate the progression of the disease.

Study Design

Study Type : Observational
Actual Enrollment : 22 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy Type II and III Treated With Valproic Acid
Study Start Date : July 2006
Primary Completion Date : August 2008
Study Completion Date : December 2008

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :
  1. Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score [ Time Frame: one year ]

Secondary Outcome Measures :
  1. Barthel Index [ Time Frame: one year ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
22 children with spinal muscular atrophy disease

Inclusion Criteria:

  • Regularly attended at the Out-patient Service of Neuromuscular Disorders and Child Neurology of our Institution;
  • More than 2 years olf and had a molecular analysis of Spinal Muscular Atrophy;

Exclusion Criteria:

  • Had been submitted into a surgery recently;
  • Did not come to the evaluation and medical sessions:
  • Did not take the medication correctly
Contacts and Locations

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 ClinicalTrials.gov identifier (NCT number): NCT01033331

Section of Neuromuscular Disorders and Service of Child Neurology, Clinics Hospital of the School of Medicine at São Paulo University
Sao PAulo, Brazil, 05403-000
Sponsors and Collaborators
University of Sao Paulo General Hospital
Principal Investigator: Umbertina Reed, Doctor Sao Paulo University
More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Evaluation of the muscle strength and motor ability in type II and III spinal muscle atrophy children treated with valproic acid, University of Sao PAulo General Hospital
ClinicalTrials.gov Identifier: NCT01033331     History of Changes
Other Study ID Numbers: 290/06
CAPES9030 ( Other Grant/Funding Number: CAPES )
First Posted: December 16, 2009    Key Record Dates
Last Update Posted: December 16, 2009
Last Verified: August 2008

Keywords provided by University of Sao Paulo General Hospital:
Spinal Muscular atrophy
Valproic acid
MRC scale

Additional relevant MeSH terms:
Muscular Atrophy
Muscular Atrophy, Spinal
Spinal Cord Diseases
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular 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