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Clinical Study of Spinal Muscular Atrophy
Not Recruiting
Trial ID: NCT00443066
Purpose
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.
Official Title
Clinical Study of Spinal Muscular Atrophy
Stanford Investigator(s)
John W. Day, MD, PhD
Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology
Eligibility
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)
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305