Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03319030 | Completed

• 1 other identifier: Aerobic Exercise DMD
• Study Type: observational
• Target: 43
• Locations: 1 country
• Sites: 1

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

exercise; Aerobic Exercise

Outcome Measures

Primary Outcomes (1)

• microRNA levels

  Understanding if there are any differences in microRNA's in boys who are more active versus those who are not.

  Baseline only

Secondary Outcomes (7)

• Physical therapy assessment - 10 meter run test

  Baseline only

• Physical therapy assessment - North Star Ambulatory Assessment

  Baseline only

• Physical therapy assessment - time to standing from supine

  Baseline only

• Cardiac Assessments: Electrocardiogram (ECG)

  Baseline only

• Cardiac Assessments: Echocardiogram (ECHO)

  Baseline only

Study Arms (1)

Duchenne Muscular Dystrophy (DMD)

Enrolls boys with a genetically confirmed diagnosis of DMD.

Eligibility Criteria

• Age: 2 Years - 17 Years
• Gender Eligibility Details: X-linked disease. Only males included.
• Age Groups: Child (0-17)
• Sampling Method: Probability Sample

Study Population

The target population is ambulatory and non-ambulatory boys with DMD.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead

Study Sites (1)

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Related Publications (7)

• 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.

  PMID: 26048046 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.

  PMID: 23440719 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.

  PMID: 25027324 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.

  PMID: 26942105 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.

  PMID: 21425469 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.

  PMID: 24436293 BACKGROUND

• 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.

  PMID: 31852847 DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

Red top blood collection to collect serum microRNA's.

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Motor Activity

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Behavior

Study Officials

• Aaron S Zelikovich

  Ann and Robert H. Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Research

Study Record Dates

• First Submitted: October 9, 2017
• First Posted: October 24, 2017
• Study Start: September 1, 2017
• Primary Completion: April 30, 2018
• Study Completion: September 30, 2018
• Last Updated: February 4, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)