Status:
COMPLETED
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
Lead Sponsor:
Carelon Research
Collaborating Sponsors:
The Spinal Muscular Atrophy Foundation
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
2-12 years
Brief 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 sev...
Detailed Description
Spinal Muscular Atrophy (SMA) is one of the two most common inherited children's neuromuscular disorders. There currently is no cure and no therapeutics approved to slow progression of the disease. SM...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 1 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
March 1 2009
Estimated Enrollment :
130 Patients enrolled
Trial Details
Trial ID
NCT00756821
Start Date
October 1 2008
End Date
March 1 2009
Last Update
October 24 2012
Active Locations (18)
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1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
Stanford University
Stanford, California, United States, 94305
3
The Children's Hospital
Aurora, Colorado, United States, 80045
4
University of Iowa
Iowa City, Iowa, United States, 52242