An observational study in Duchenne Muscular Dystrophy, sponsored by Ann & Robert H Lurie Children's Hospital of Chicago. Completed at 1 site in United States. Open to male participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2019-02-04.
Sponsored by Ann & Robert H Lurie Children's Hospital of Chicago · Observational
Among 232 studies in Muscular Dystrophy, Duchenne with a site in United States — 36 open to participants now, 60 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · United States.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, this one is the 41st largest by enrollment of the 55 observational ones that give a count, is one of 11 that started in 2017, is one of 55 observational ones, and was the 116th registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →36 of the 232 studies in Muscular Dystrophy, Duchenne with a site in United States are open now. Recruiting first, the newest first:
Sarepta Therapeutics, Inc. · Sites in United States
University of Florida · Sites in United States
Vanderbilt University Medical Center · Sites in United States
University of Rochester · Sites in United States
Dyne Therapeutics · Sites in United States
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 60 take children under 18; 12 take healthy volunteers.
This study: 2 Years to 17 Years · male.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
232 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 57 · Phase 2 90 · Phase 3 40 · Phase 4 5
First in 2000; this study’s year is marked. 11 started in 2017.
This study's enrollment of 43 is below the median of 60 across 132 observational studies indexed under Muscular Dystrophy, Duchenne.
Median enrollment 25 across the 171 interventional studies, 75 across the 55 observational. Of the 160 that completed or were terminated, 77 (48%) have results posted.
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130 of 232 have an industry lead sponsor. The 10 most frequent:
Ann & Robert H Lurie Children's Hospital of Chicago is the lead sponsor of 180 studies on the registry; 28 are open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 5 (50%) have results posted.
Ann & Robert H Lurie Children's Hospital of Chicago has 2 studies filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
19 of the studies in Muscular Dystrophy, Duchenne with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT06290713University of FloridaVasodilator and Exercise Study for DMD (VASO-REx) | 2026-11 |
| NCT06450639Hoffmann-La RocheA Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) | 2026-11-18 |
| NCT03368742Solid Biosciences Inc.Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | 2026-11-30 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, from their registry updates:
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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.
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.
The target population is ambulatory and non-ambulatory boys with DMD.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Enrolls boys with a genetically confirmed diagnosis of DMD.
microRNA levels
Understanding if there are any differences in microRNA's in boys who are more active versus those who are not.
Time frame: Baseline only
Physical therapy assessment - 10 meter run test
Time in seconds that it takes a participant to run 10 meters.
Time frame: Baseline only
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.
Time frame: Baseline only
Physical therapy assessment - time to standing from supine
Time in seconds that it takes a participant to stand from a supine position.
Time frame: Baseline only
Cardiac Assessments: Electrocardiogram (ECG)
Comparing heart rate (BPM), PR interval (msec), QRS (msec), and QT intervals (msec) to microRNA levels.
Time frame: Baseline only
Cardiac Assessments: Echocardiogram (ECHO)
Comparing ejection fraction (%) and other Left Ventricle systolic and diastolic metrics (cm) and microRNA levels.
Time frame: Baseline only
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.
Time frame: Baseline only
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.
Time frame: Baseline only
Plan to share: No
Ran from Sep 2017 to primary completion in Apr 2018, 7 months.
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This study is completed, as verified in Jan 2019. You cannot join it, but the record below documents what was studied.
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Muscular Dystrophy, Duchenne →
Ann & Robert H Lurie Children's Hospital of Chicago →