NCT01033331

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
22

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jul 2006

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2006

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2008

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2008

Completed
1 year until next milestone

First Submitted

Initial submission to the registry

December 15, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

December 16, 2009

Completed
Last Updated

December 16, 2009

Status Verified

August 1, 2008

Enrollment Period

2.1 years

First QC Date

December 15, 2009

Last Update Submit

December 15, 2009

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular atrophyValproic acidHMASMRC scale

Outcome Measures

Primary Outcomes (1)

  • Manual Muscle Test (Medical Research Council scale-MRC), the Hammersmith Motor Ability Score

    one year

Secondary Outcomes (1)

  • Barthel Index

    one year

Eligibility Criteria

Age2 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

22 children with spinal muscular atrophy disease

You may qualify if:

  • 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;

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Section of Neuromuscular Disorders and Service of Child Neurology, Clinics Hospital of the School of Medicine at São Paulo University

São Paulo, São Paulo, 05403-000, Brazil

Location

Related Publications (2)

  • 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 atrophy. PLoS One. 2009;4(5):e5268. doi: 10.1371/journal.pone.0005268. Epub 2009 May 14.

    PMID: 19440247RESULT

  • Darbar IA, Plaggert PG, Resende MB, Zanoteli E, Reed UC. Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid. BMC Neurol. 2011 Mar 24;11:36. doi: 10.1186/1471-2377-11-36.

    PMID: 21435220DERIVED

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Umbertina Reed, Doctor

    Sao Paulo University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER

Study Record Dates

First Submitted

December 15, 2009

First Posted

December 16, 2009

Study Start

July 1, 2006

Primary Completion

August 1, 2008

Study Completion

December 1, 2008

Last Updated

December 16, 2009

Record last verified: 2008-08

Locations

BR(1)