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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Duchenne Muscular Dystrophy (DMD) | Enrolls boys with a genetically confirmed diagnosis of DMD. |
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| Measure | Description | Time Frame |
|---|---|---|
| microRNA levels | Understanding if there are any differences in microRNA's in boys who are more active versus those who are not. | Baseline only |
| Measure | Description | Time Frame |
|---|---|---|
| Physical therapy assessment - 10 meter run test | Time in seconds that it takes a participant to run 10 meters. | Baseline only |
| Physical therapy assessment - North Star Ambulatory Assessment | Comparing a standardized test for ambulatory boys with DMD that gives a score out of 34 total points and microRNA levels. |
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Inclusion Criteria:
Exclusion Criteria:
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X-linked disease. Only males included.
The target population is ambulatory and non-ambulatory boys with DMD.
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| Name | Affiliation | Role |
|---|---|---|
| Aaron S Zelikovich | Ann & Robert H Lurie Children's Hospital of Chicago | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Ann and Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26048046 | Background | Guiraud S, Aartsma-Rus A, Vieira NM, Davies KE, van Ommen GJ, Kunkel LM. The Pathogenesis and Therapy of Muscular Dystrophies. Annu Rev Genomics Hum Genet. 2015;16:281-308. doi: 10.1146/annurev-genom-090314-025003. Epub 2015 Jun 4. | |
| 23440719 | Background | Flanigan KM, Ceco E, Lamar KM, Kaminoh Y, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Gappmaier E, Howard MT, Day JW, McDonald C, McNally EM, Weiss RB; United Dystrophinopathy Project. LTBP4 genotype predicts age of ambulatory loss in Duchenne muscular dystrophy. Ann Neurol. 2013 Apr;73(4):481-8. doi: 10.1002/ana.23819. Epub 2013 Feb 20. |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009043 | Motor Activity |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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Red top blood collection to collect serum microRNA's.
| Baseline only |
| Physical therapy assessment - time to standing from supine | Time in seconds that it takes a participant to stand from a supine position. | Baseline only |
| Cardiac Assessments: Electrocardiogram (ECG) | Comparing heart rate (BPM), PR interval (msec), QRS (msec), and QT intervals (msec) to microRNA levels. | Baseline only |
| Cardiac Assessments: Echocardiogram (ECHO) | Comparing ejection fraction (%) and other Left Ventricle systolic and diastolic metrics (cm) and microRNA levels. | Baseline only |
| Questionnaire: Pediatric Quality of Life: Neuromuscular module | 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. | Baseline only |
| Questionnaire: Physical Function Survey | 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. | Baseline only |
| 25027324 | Background | Hathout Y, Marathi RL, Rayavarapu S, Zhang A, Brown KJ, Seol H, Gordish-Dressman H, Cirak S, Bello L, Nagaraju K, Partridge T, Hoffman EP, Takeda S, Mah JK, Henricson E, McDonald C. Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients. Hum Mol Genet. 2014 Dec 15;23(24):6458-69. doi: 10.1093/hmg/ddu366. Epub 2014 Jul 15. |
| 26942105 | Background | Giordani L, Sandona M, Rotini A, Puri PL, Consalvi S, Saccone V. Muscle-specific microRNAs as biomarkers of Duchenne Muscular Dystrophy progression and response to therapies. Rare Dis. 2014 Dec 1;2(1):e974969. doi: 10.4161/21675511.2014.974969. eCollection 2014. |
| 21425469 | Background | Cacchiarelli D, Legnini I, Martone J, Cazzella V, D'Amico A, Bertini E, Bozzoni I. miRNAs as serum biomarkers for Duchenne muscular dystrophy. EMBO Mol Med. 2011 May;3(5):258-65. doi: 10.1002/emmm.201100133. Epub 2011 Mar 21. |
| 24436293 | Background | Baggish AL, Park J, Min PK, Isaacs S, Parker BA, Thompson PD, Troyanos C, D'Hemecourt P, Dyer S, Thiel M, Hale A, Chan SY. Rapid upregulation and clearance of distinct circulating microRNAs after prolonged aerobic exercise. J Appl Physiol (1985). 2014 Mar 1;116(5):522-31. doi: 10.1152/japplphysiol.01141.2013. Epub 2014 Jan 16. |
| 31852847 | Derived | Quattrocelli M, Zelikovich AS, Jiang Z, Peek CB, Demonbreun AR, Kuntz NL, Barish GD, Haldar SM, Bass J, McNally EM. Pulsed glucocorticoids enhance dystrophic muscle performance through epigenetic-metabolic reprogramming. JCI Insight. 2019 Dec 19;4(24):e132402. doi: 10.1172/jci.insight.132402. |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D001519 | Behavior |