Clinical Study of Spinal Muscular Atrophy

Completed

Conditions studied: Spinal Muscular Atrophy

In brief

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.

Key facts

Study ID: NCT00443066
Run by: Columbia University
People needed: 120
Starts: 2005-05-01
Expected to finish: 2011-08-17
Last updated by the study team: 2023-03-21

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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)

Where it is running

Columbia University SMA Clinical Research Center — New York, New York, United States

Full record on ClinicalTrials.gov

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