This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec (ELEVIDYS) as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at the time of study enrollment in routine clinical practice.
Study Type
OBSERVATIONAL
Enrollment
500
No study medication will be provided by the sponsor during this study.
No study medication will be provided by the sponsor during this study.
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles - PIN
Los Angeles, California, United States
University of Colorado - PPDS
Aurora, Colorado, United States
Connecticut Children's Medical Center - Hartford
Hartford, Connecticut, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12
Time frame: Baseline, Month 12
Number of Participants Experiencing Acute Liver Injury (ALI)
Time frame: Baseline through Month 12
Time to Rise From Floor (Supine to Stand)
Time frame: Up to 10 years
Loss of Ambulation (LOA)
Time frame: Up to 10 years
Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score
Time frame: Up to 10 years
Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue
Time frame: Up to 10 years
Pulmonary Function as Measured by Forced Vital Capacity (FVC)
Time frame: Up to 10 years
Pulmonary Function as Measured by FVC Percent Predicted (FVC%p)
Time frame: Up to 10 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI)
Time frame: Up to 10 years
Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF)
Time frame: Baseline through Month 12
Number of Participants Experiencing Complications Associated with ALI and ALF
Time frame: Up to 10 years
Survival Time
Time frame: Up to 10 years
Time to First Vertebral Body (Spine) Fracture
Time frame: Up to 10 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Nicklaus Children's Hospital
Miami, Florida, United States
Nemours Children's Hospital - Orlando
Orlando, Florida, United States
All Children's Research Institute, Inc
St. Petersburg, Florida, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States
Indiana Clinical and Translational Science Institute
Indianapolis, Indiana, United States
...and 15 more locations