Status:

COMPLETED

Clinical Study of Spinal Muscular Atrophy

Lead Sponsor:

Columbia University

Collaborating Sponsors:

The Spinal Muscular Atrophy Foundation

Conditions:

Spinal Muscular Atrophy

Eligibility:

All Genders

Brief Summary

The investigators propose to prepare for clinical trials where SMA patients are asked to join the research effort. The visits will include questions, physical exam, blood drawing, and sometimes X-rays...

Detailed Description

Spinal Muscular Atrophy (SMA) is one of the most devastating neurological diseases of childhood. Affected infants and children suffer from progressive muscle weakness caused by degeneration of lower m...

Eligibility Criteria

Inclusion

  • Inclusion Criteria
  • Clinical diagnosis of Spinal Muscular Atrophy
  • Genetic diagnosis of SMN gene deletion
  • Parents or if applicable subjects must give informed consent
  • must be capable of complying with the study procedures
  • Female subjects of child-bearing potential must agree to undergo pregnancy test prior to radiological studies
  • Diagnosis of SMA before age 19 years
  • Exclusion Criteria:
  • Unstable medical condition precluding participation
  • Significant respiratory compromise that would interfere with safe travel to site of evaluation. (The clinical site PI decides when air travel is not recommended and when the patient's location is not within a reasonably safe driving distance (upper limit 150-250 miles)

Exclusion

    Key Trial Info

    Start Date :

    May 1 2005

    Trial Type :

    OBSERVATIONAL

    Allocation :

    ACTUAL

    End Date :

    August 17 2011

    Estimated Enrollment :

    120 Patients enrolled

    Trial Details

    Trial ID

    NCT00443066

    Start Date

    May 1 2005

    End Date

    August 17 2011

    Last Update

    March 21 2023

    Active Locations (1)

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    Page 1 of 1 (1 locations)

    1

    Columbia University SMA Clinical Research Center

    New York, New York, United States, 10032