Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
This study is being conducted to test whether exercise can be effectively used as an intervention to treat Spinal Muscular Atrophy (SMA). In order to answer this question, the investigators will enroll 14 subjects with SMA between ages 8 and 50 and ask them to complete an 18 month training schedule. At some points subjects will be asked to closely follow a specific training regimen and at other points they may be asked to exercise in the same manner they do normally. The exercises they will be asked to perform include biking on a stationary cycle and lifting hand weights. Subjects will be asked to come in to the clinic seven times over the course of the study to perform tests. These tests include motor function measures, a physical exam, questionnaires, a exercise capacity test which involves riding a stationary bicycle, and test where the subject is asked to walk as far as they can in six minutes. The main goal of the study is to see if the subjects who participate in the exercise protocol have larger increases in the distance they can walk in six minutes than those who do not.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Randomized, Controlled Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)|
- Distance walked in the Six Minute Walk Test (6MWT). [ Time Frame: Up to 19 months ] [ Designated as safety issue: No ]
|Study Start Date:||November 2010|
|Estimated Study Completion Date:||August 2014|
|Primary Completion Date:||February 2013 (Final data collection date for primary outcome measure)|
Muscle strengthening program using weights and resistance bands in combination with a home based cycle ergometry program. The home-based exercise program will be performed up to 5 times weekly.
Muscle strengthening program using hand weights and resistance bands in combination with a home based cycle ergometry. This home-based exercise program will be performed up to 5 times weekly. Each session will consist of up to 30 minutes of cycling and 30 minutes of strengthening.
No Intervention: Typical Activity
Subjects in this group will be asked to maintain their typical daily activity. Those assigned to this arm will be given the opportunity to join the intervention arm seven months after their screening visit.
|United States, New York|
|Columbia University SMA Clinical Research Center|
|New York, New York, United States, 10032|
|Principal Investigator:||Darryl C De Vivo, MD||Columbia University|