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

Sponsor

University of Sao Paulo General Hospital (Other)

Overall Status

Completed

CT.gov ID

NCT01033331

Collaborator

(none)

Enrollment

Location

Duration (Months)

0.8

Patients Per Site Per Month

Study Details

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	Intervention/Treatment	Phase
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 :

Jul 1, 2006

Actual Primary Completion Date :

Aug 1, 2008

Actual Study Completion Date :

Dec 1, 2008

Outcome Measures

Primary Outcome Measures

Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score [one year]

Secondary Outcome Measures

Barthel Index [one year]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Locations

	Site	City	State	Country	Postal Code
1	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