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

Spinal Muscular Atrophy Clinical Trial

Official title:

Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy Type II and III Treated With Valproic Acid

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01033331
• Other study ID #: 290/06
• Secondary ID: CAPES9030
• Status: Completed
• Phase: N/A
• First received: December 15, 2009
• Last updated: December 15, 2009
• Start date: July 2006
• Est. completion date: December 2008

Study information

• Verified date: August 2008
• Source: University of Sao Paulo General Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: Brazil: Ethics Committee
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: December 2008
• Est. primary completion date: August 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Years to 18 Years

Eligibility

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

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy

Locations

Country	Name	City	State
Brazil	Section of Neuromuscular Disorders and Service of Child Neurology, Clinics Hospital of the School of Medicine at São Paulo University	Sao PAulo

Sponsors (1)

Lead Sponsor	Collaborator
University of Sao Paulo General Hospital

Country where clinical trial is conducted

Brazil, 

References & Publications (1)

Swoboda KJ, Scott CB, Reyna SP, Prior TW, LaSalle B, Sorenson SL, Wood J, Acsadi G, Crawford TO, Kissel JT, Krosschell KJ, D'Anjou G, Bromberg MB, Schroth MK, Chan GM, Elsheikh B, Simard LR. Phase II open label study of valproic acid in spinal muscular at — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score		one year	Yes
Secondary	Barthel Index		one year	Yes