Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

NCT ID: NCT03319030

Last Updated: 2019-02-04

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

43 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-09-01

Study Completion Date

2018-09-30

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Duchenne Muscular Dystrophy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Duchenne Muscular Dystrophy (DMD)

Enrolls boys with a genetically confirmed diagnosis of DMD.

No interventions assigned to this group

Eligibility Criteria

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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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

17 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role lead

Responsible Party

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Aaron Zelikovich

Clinical Research

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Aaron S Zelikovich

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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

Chicago, Illinois, United States

Site Status

Countries

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United States

References

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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.

Reference Type BACKGROUND
PMID: 26048046 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 23440719 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 25027324 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 26942105 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 21425469 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 24436293 (View on PubMed)

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.

Reference Type DERIVED
PMID: 31852847 (View on PubMed)

Other Identifiers

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Aerobic Exercise DMD

Identifier Type: -

Identifier Source: org_study_id

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