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Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT03319030
Recruitment Status : Enrolling by invitation
First Posted : October 24, 2017
Last Update Posted : October 24, 2017
Sponsor:
Information provided by (Responsible Party):
Aaron Zelikovich, Ann & Robert H Lurie Children's Hospital of Chicago

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.

Condition or disease
Duchenne Muscular Dystrophy

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.


Study Type : Observational
Estimated Enrollment : 55 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Evaluating the Impact of Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
Actual Study Start Date : September 1, 2017
Estimated Primary Completion Date : April 2018
Estimated Study Completion Date : September 2018


Group/Cohort
Duchenne Muscular Dystrophy (DMD)
Enrolls boys with a genetically confirmed diagnosis of DMD.



Primary Outcome Measures :
  1. microRNA levels [ Time Frame: Baseline only ]
    Understanding if there are any differences in microRNA's in boys who are more active versus those who are not.


Secondary Outcome Measures :
  1. Physical therapy assessment - 10 meter run test [ Time Frame: Baseline only ]
    Time in seconds that it takes a participant to run 10 meters.

  2. Physical therapy assessment - North Star Ambulatory Assessment [ Time Frame: Baseline only ]
    Comparing a standardized test for ambulatory boys with DMD that gives a score out of 34 total points and microRNA levels.

  3. Physical therapy assessment - time to standing from supine [ Time Frame: Baseline only ]
    Time in seconds that it takes a participant to stand from a supine position.

  4. Cardiac Assessments: Electrocardiogram (ECG) [ Time Frame: Baseline only ]
    Comparing heart rate (BPM), PR interval (msec), QRS (msec), and QT intervals (msec) to microRNA levels.

  5. Cardiac Assessments: Echocardiogram (ECHO) [ Time Frame: Baseline only ]
    Comparing ejection fraction (%) and other Left Ventricle systolic and diastolic metrics (cm) and microRNA levels.

  6. Questionnaire: Pediatric Quality of Life: Neuromuscular module [ Time Frame: Baseline only ]
    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.

  7. Questionnaire: Physical Function Survey [ Time Frame: Baseline only ]
    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.


Biospecimen Retention:   Samples Without DNA
Red top blood collection to collect serum microRNA's.


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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   X-linked disease. Only males included.
Sampling Method:   Probability Sample
Study Population
The target population is ambulatory and non-ambulatory boys with DMD.
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03319030


Locations
United States, Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Sponsors and Collaborators
Ann & Robert H Lurie Children's Hospital of Chicago
Investigators
Principal Investigator: Aaron S Zelikovich Ann and Robert H. Lurie Children's Hospital of Chicago

Publications:

Responsible Party: Aaron Zelikovich, Clinical Research, Ann & Robert H Lurie Children's Hospital of Chicago
ClinicalTrials.gov Identifier: NCT03319030     History of Changes
Other Study ID Numbers: Aerobic Exercise DMD
First Posted: October 24, 2017    Key Record Dates
Last Update Posted: October 24, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Aaron Zelikovich, Ann & Robert H Lurie Children's Hospital of Chicago:
exercise
Aerobic Exercise

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked