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Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
study id #: NCT03319030
condition: Duchenne Muscular Dystrophy
This research study wants to learn more about Duchenne Muscular Dystrophy (DMD) and exercise. Today it is unknown how exercising impacts boys with DMD. The investigators believe that increasing activity and aerobic exercise may help with heart, lung, and muscle function. The investigators are hoping to compare physical strength and blood samples of boys with DMD to see if there are any differences between kids who exercised more as a child versus those who didn’t.
mechanism of action: No pharmaceutical intervention
last updated: February 15, 2019
start date: September 1, 2017
estimated completion: September 30, 2018
size / enrollment: 43
Recently, the American Academy of Neurology released new guidelines for children with Duchenne Muscular Dystrophy (DMD) with an updated recommendation to include moderate aerobic exercise. Historically, children with DMD were advised to not exercise as it was thought to accelerate muscle loss. Today, there is new data suggesting that moderate aerobic exercise that does not strain muscle may be beneficial and improve a child's quality of life with regard to heart disease, fatigue, and other factors of overall health. Although it is expected that children who exercise will experience health benefits, there is no scientific evidence indicating a positive or negative effect in a DMD population.
This study is trying to understand the role of aerobic exercise in boys with DMD and how it may impact heart, muscle, and lung function using newly identified biomarkers. The goal is to correlate increased aerobic exercise with improved functional outcomes and changes in biomarkers.
The data collected from this study will allow future development and implementation of an innovative and disease-specific home aerobic exercise program that in the future will be recommended to families of children with DMD.
- microRNA levels [Time Frame: Baseline only]
Understanding if there are any differences in microRNA's in boys who are more active versus those who are not.
- Physical therapy assessment - 10 meter run test [Time Frame: Baseline only]
Time in seconds that it takes a participant to run 10 meters.
- Physical therapy assessment - North Star Ambulatory Assessment [Time Frame: Baseline only]
Comparing a standardized test for ambulatory boys with DMD that gives a score out of 34 total points and microRNA levels.
- Physical therapy assessment - time to standing from supine [Time Frame: Baseline only]
Time in seconds that it takes a participant to stand from a supine position.
- Cardiac Assessments: Electrocardiogram (ECG) [Time Frame: Baseline only]
Comparing heart rate (BPM), PR interval (msec), QRS (msec), and QT intervals (msec) to microRNA levels.
- Cardiac Assessments: Echocardiogram (ECHO) [Time Frame: Baseline only]
Comparing ejection fraction (%) and other Left Ventricle systolic and diastolic metrics (cm) and microRNA levels.
- Questionnaire: Pediatric Quality of Life: Neuromuscular module [Time Frame: Baseline only]
To see if there is any correlation between Quality of Life scores (range from 0 to 100) and microRNA levels. Increased scores indicate a higher quality of life.
- Questionnaire: Physical Function Survey [Time Frame: Baseline only]
To see if there is any correlation between scores of functional ability (with a range from 0 to 106) and microRNA levels. Higher physical function function scores indicate increased strength.
Ages 2-17 years old with a confirmed diagnosis of DMD. Has an appointment in MDA clinic at the Ann & Robert H. Lurie Children's Hospital of Chicago.
Is less than two years old and does not have a confirmed diagnosis of DMD.
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