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

This study has been completed.
Sponsor:
Information provided by:
University of Sao Paulo General Hospital
ClinicalTrials.gov Identifier:
NCT01033331
First received: December 15, 2009
Last updated: NA
Last verified: August 2008
History: No changes posted
  Purpose

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
Spinal Muscular Atrophy

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by University of Sao Paulo General Hospital:

Primary Outcome Measures:
  • Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score [ Time Frame: one year ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Barthel Index [ Time Frame: one year ] [ Designated as safety issue: Yes ]

Enrollment: 22
Study Start Date: July 2006
Study Completion Date: December 2008
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
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.

  Eligibility

Ages Eligible for Study:   2 Years to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

22 children with spinal muscular atrophy disease

Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01033331

Locations
Brazil
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
Investigators
Principal Investigator: Umbertina Reed, Doctor Sao Paulo University
  More Information

Publications:
Additional 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
Study First Received: December 15, 2009
Last Updated: December 15, 2009
Health Authority: Brazil: Ethics Committee

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

Additional relevant MeSH terms:
Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathological Conditions, Anatomical
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Valproic Acid
Anticonvulsants
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
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

ClinicalTrials.gov processed this record on August 18, 2014