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Duchenne Muscular DystrophyNCT03319030

Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

An observational study of Duchenne Muscular Dystrophy, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 17 years, men only
Conditions the study is for
Duchenne Muscular Dystrophy
How many people
43 participants
Where
1 siteUnited States
Study dates
1 Sep 2017to 30 Sep 2018
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 17 years
  • Men only
  • Child

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

Who may be able to join

  • 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.

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

United States1 site

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

    Chicago, Illinois, 60611

Other trials in United States

About this study

Official title: Evaluating the Impact of Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

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.

Read the 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.

Keywords
exercise, Aerobic Exercise
Medical subject headings
Muscular Dystrophy, Duchenne, Motor Activity

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT03319030
Study groupRoleWhat they receive
Duchenne Muscular Dystrophy (DMD)Enrolls boys with a genetically confirmed diagnosis of DMD.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. microRNA levels

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

    Measured: Baseline only

Secondary outcomes (7)

  1. Physical therapy assessment - 10 meter run test

    Time in seconds that it takes a participant to run 10 meters.

    Measured: Baseline only

  2. 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.

    Measured: Baseline only

  3. Physical therapy assessment - time to standing from supine

    Time in seconds that it takes a participant to stand from a supine position.

    Measured: Baseline only

  4. Cardiac Assessments: Electrocardiogram (ECG)

    Comparing heart rate (BPM), PR interval (msec), QRS (msec), and QT intervals (msec) to microRNA levels.

    Measured: Baseline only

  5. Cardiac Assessments: Echocardiogram (ECHO)

    Comparing ejection fraction (%) and other Left Ventricle systolic and diastolic metrics (cm) and microRNA levels.

    Measured: Baseline only

  6. 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.

    Measured: Baseline only

  7. 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.

    Measured: Baseline only

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Oct 2017
  2. Study start1 Sep 2017
  3. Primary completion30 Apr 2018
  4. Study completion30 Sep 2018

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Cross-sectional
Enrolment
43 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03319030
Sponsor's protocol ID
Aerobic Exercise DMD
Status last verified
Jan 2019
First submitted
9 Oct 2017
Last updated
4 Feb 2019
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 26048046

  • 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.

    PubMed 23440719

  • 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.

    PubMed 25027324

  • 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.

    PubMed 26942105

  • 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.

    PubMed 21425469

  • 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.

    PubMed 24436293

  • 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.

    PubMed 31852847

Questions about this trial

What is this clinical trial studying?

An observational study of Duchenne Muscular Dystrophy, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 4 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

microRNA levels (time frame: Baseline only). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03319030, which also lists 7 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Feb 2019.

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