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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 (Treated) | Cohort 1: participants prescribed ELEVIDYS in a commercial setting. Cohort 1a (Ambulatory ELEVIDYS Prospectively Treated Cohort): participants prescribed ELEVIDYS by commercially treating physicians with consent no later than 30 days from infusion. All participants will be dosed with ELEVIDYS based on United States prescribing information (USPI). The Post Market Requirement (PMR) Cohort (sub-cohort of 1a) consists of participants with laboratory data as specified in the ELEVIDYS USPI. Cohort 1b (Non-ambulatory ELEVIDYS Prospectively Treated Cohort): non-ambulatory DMD participants prescribed ELEVIDYS commercially and recruited by treating physicians before infusion (enrollment currently closed). Cohort 1c (ELEVIDYS Retrospectively Treated Cohort): participants dosed with ELEVIDYS with complete baseline data within 6 months prior to dosing and complete prospectively collected annual follow-up data after infusion until the time of cohort entry (sponsor approval required for enrollment). |
| |
| Cohort 2 (Standard of Care) | Cohort 2: ambulatory DMD participants who are at least 4 years of age at baseline, unexposed to DMD gene therapy, and receiving or prescribed chronic glucocorticoids at study entry. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Delandistrogene Moxeparvovec | Genetic | No study medication will be provided by the sponsor during this study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change From Baseline in Time to Walk/Run 10 Meters (10MWR) (Calculated Velocity) at Month 12 | Baseline, Month 12 | |
| Number of Participants Experiencing Acute Liver Injury (ALI) | Baseline through Month 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Rise From Floor (Supine to Stand) | Up to 10 years | |
| Loss of Ambulation (LOA) | Up to 10 years | |
| Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score |
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Inclusion Criteria:
For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):
For Standard of Care Comparators (Cohort 2):
Exclusion Criteria:
Has any deletion of exon 8 and/or exon 9 in the DMD gene.
Is currently participating in any DMD interventional study at the time of this observational study enrollment.
Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).
Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:
Other inclusion/exclusion criteria may apply.
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DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Children's Hospital Los Angeles - PIN |
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| Standard of Care | Drug | No study medication will be provided by the sponsor during this study. |
|
|
| Up to 10 years |
| Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue | Up to 10 years |
| Pulmonary Function as Measured by Forced Vital Capacity (FVC) | Up to 10 years |
| Pulmonary Function as Measured by FVC Percent Predicted (FVC%p) | Up to 10 years |
| Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI) | Up to 10 years |
| Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF) | Baseline through Month 12 |
| Number of Participants Experiencing Complications Associated with ALI and ALF | Up to 10 years |
| Survival Time | Up to 10 years |
| Time to First Vertebral Body (Spine) Fracture | Up to 10 years |
| Los Angeles |
| California |
| 90027 |
| United States |
| University of Colorado - PPDS | Aurora | Colorado | 80045 | United States |
| Connecticut Children's Medical Center - Hartford | Hartford | Connecticut | 06106 | United States |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | United States |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States |
| Nemours Children's Hospital - Orlando | Orlando | Florida | 32827 | United States |
| All Children's Research Institute, Inc | St. Petersburg | Florida | 33701 | United States |
| Ann and Robert H Lurie Childrens Hospital of Chicago | Chicago | Illinois | 60611-2991 | United States |
| Indiana Clinical and Translational Science Institute | Indianapolis | Indiana | 46202 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Duke Lenox Baker Children's Hospital | Durham | North Carolina | 27704 | United States |
| Childrens Hospital Medical Center of Akron | Akron | Ohio | 44302 | United States |
| OHSU Healthcare (Oregon Health and Science University) | Portland | Oregon | 97239 | United States |
| Penn State Health Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Le Bonheur Children's Hospital | Memphis | Tennessee | 38103 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| Cook Children's Hospital | Fort Worth | Texas | 76104 | United States |
| University of Virginia | Charlottesville | Virginia | 22903 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| The Board of Regents of the University of Wisconsin | Madison | Wisconsin | 53715-1218 | United States |
| The Medical College of Wisconsin | Milwaukee | Wisconsin | 53226-4874 | United States |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D059039 | Standard of Care |
| ID | Term |
|---|---|
| D019984 | Quality Indicators, Health Care |
| D011787 | Quality of Health Care |
| D006298 | Health Services Administration |
| D017530 | Health Care Quality, Access, and Evaluation |
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