Clinical trial NCT03319030: Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Official title: Evaluating the Impact of Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Duchenne Muscular Dystrophy
Keywords: exercise
Aerobic Exercise
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Sponsor class: OTHER
First posted: 2017-10-24
Enrollment: 43
Sex: MALE
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: PROBABILITY_SAMPLE
Study population: The target population is ambulatory and non-ambulatory boys with DMD.

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* Is less than two years old and does not have a confirmed diagnosis of DMD.

Primary outcomes:
measure: microRNA levels
timeFrame: Baseline only
Secondary outcomes:
measure: Physical therapy assessment - 10 meter run test
timeFrame: Baseline only
measure: Physical therapy assessment - North Star Ambulatory Assessment
timeFrame: Baseline only
measure: Physical therapy assessment - time to standing from supine
timeFrame: Baseline only
measure: Cardiac Assessments: Electrocardiogram (ECG)
timeFrame: Baseline only
measure: Cardiac Assessments: Echocardiogram (ECHO)
timeFrame: Baseline only
measure: Questionnaire: Pediatric Quality of Life: Neuromuscular module
timeFrame: Baseline only
measure: Questionnaire: Physical Function Survey
timeFrame: Baseline only
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
Study officials:
affiliation: Ann and Robert H. Lurie Children's Hospital of Chicago
name: Aaron S Zelikovich
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.