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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
Sponsor:
Information provided by:
University of Sao Paulo General Hospital

December 15, 2009
December 16, 2009
December 16, 2009
July 2006
August 2008   (Final data collection date for primary outcome measure)
Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score [ Time Frame: one year ]
Same as current
No Changes Posted
Barthel Index [ Time Frame: one year ]
Same as current
Not Provided
Not Provided
 
Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With Valproic Acid
Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy Type II and III Treated With Valproic Acid
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.
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.
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Non-Probability Sample
22 children with spinal muscular atrophy disease
Spinal Muscular Atrophy
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
22
Same as current
December 2008
August 2008   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
2 Years to 18 Years   (Child, Adult)
No
Contact information is only displayed when the study is recruiting subjects
Brazil
 
 
NCT01033331
290/06
CAPES9030 ( Other Grant/Funding Number: CAPES )
Yes
Not Provided
Not Provided
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
University of Sao Paulo General Hospital
Not Provided
Principal Investigator: Umbertina Reed, Doctor Sao Paulo University
University of Sao Paulo General Hospital
August 2008