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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1
Columbia University SMA Clinical Research Center
New York, New York, United States, 10032