A Pilot Study of Biomarkers for Spinal Muscular Atrophy

Study Identifier:
BforSMA
ClinicalTrials.gov Identifier:
NCT00756821
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Carelon Research
Study Complete

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
Study Drug
    Date
    Oct 2008 - Mar 2009
    Patient Requirements
    Sex: Female & Male
    Age: 2 - 12 Years
    Requirements Information
    Inclusion and Exclusion Criteria
    Inclusion Criteria
    • Age 2 to 12 years, inclusive
    • In good health (other than SMA) in the judgement of the clinical investigator ar the time of assessment
    Exclusion Criteria
    • Systemic or specific-organ illness
    • Any known genetic condition other than SMA requiring pharmaceutical treatment
    • Use of any putative SMN-enhancing medications or treatments in the past 14 days prior to enrollment
    • Use of carnitine, creatine, oral albuterol or riluzole for 14 days prior to enrollment
    • Use of any oral prescription medications for 14 days prior to enrollment (exceptions: anti-reflux medications, constipation or stoll softening medications, stool bulking agents, and inhaled bronchodilator medications)
    • Any illness requiring treatment of antibiotics or anti-inflammatory medication within the past 14 days
    • Any rash requiring treatment within the past 7 days
    • Any severe asthma attack requiring treatment with oral or parenteral steroids within the past 7 days
    • Any fever over 100 degrees Fahrenheit or 38 degree Celsius within the past 7 days
    • Any immunization within the past 7 days
    • Any injury sustained that resulted in a bone fracture or needed stitches within the past 7 days
    • Any surgery within the past 7 days
    • Any receipt of anesthesia within the past 7 days
    • Any Emergency Room visit or hospitalization within the past 7 days
    • Any stomach illness with vomiting within the past 7 days
    • Any migraine headache within the past 7 days
    • Participation in a clinical trial (except observational studies) within the past 7 days

    Protocol Summary

    The goal of this pilot study is to identify a marker or panel of markers in the blood or urine from a wide range of Spinal Muscular Atrophy (SMA) patients that segregates with measures of clinical severity. From this identification of candidate biomarkers, it is hoped that further investigations, both longitudinal natural history and clinical efficacy studies, will verify a biomarker with the sensitivity and specificity that will allow its eventual use as a validated pharmacodynamic marker or surrogate endpoint. In addition, this effort may elucidate biological pathways that may be potential therapeutic targets.

    Trial Locations

    Location
    Status
    Location
    University of Alabama at Birmingham
    Birmingham, Alabama, United States, 35233
    Status
    N/A
    Location
    Stanford University
    Stanford, California, United States, 94305
    Status
    N/A
    Location
    The Children's Hospital
    Aurora, Colorado, United States, 80045
    Status
    N/A
    Location
    University of Iowa
    Iowa City, Iowa, United States, 52242
    Status
    N/A
    Location
    Johns Hopkins Hospital
    Baltimore, Maryland, United States, 21287
    Status
    N/A
    Location
    Children's Hospital Boston
    Boston, Massachusetts, United States, 02115
    Status
    N/A
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