Clinical Study of Spinal Muscular Atrophy

Study Identifier:
AAAB1635
ClinicalTrials.gov Identifier:
NCT00443066
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Columbia University
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    May 2005 - Aug 2011
    Patient Requirements
    Sex: Female & Male
    Age: N/A
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • 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)

    Protocol 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 and a skin biopsy. The investigators will use modern computer methods to process the information during which the investigators will plan a clinical trial. Once the clinical trial begins, the investigators will offer SMA patients participation if they meet the criteria for that trial.

    Identifying an effective SMA treatment is very important because there is currently none. Clinical trials are the only way to decide whether a new treatment works in SMA patients or not.

    Trial Locations

    Location
    Status
    Location
    Columbia University SMA Clinical Research Center
    New York, New York, United States, 10032
    Status
    N/A

    Contact Cure SMA

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