--- title: An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy nct_id: NCT06270719 overall_status: ENROLLING_BY_INVITATION sponsor: Sarepta Therapeutics, Inc. study_type: OBSERVATIONAL primary_condition: Duchenne Muscular Dystrophy countries: United States canonical_url: "https://parkinsonspathways.com/agent/trials/NCT06270719.md" clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT06270719" ct_last_update_post_date: 2026-05-04 last_seen_at: "2026-05-12T06:30:34.684Z" source: ClinicalTrials.gov (mirrored, no enrichment) --- # An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy **Official Title:** A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice **NCT ID:** [NCT06270719](https://clinicaltrials.gov/study/NCT06270719) ## Key Facts - **Status:** ENROLLING_BY_INVITATION - **Study Type:** OBSERVATIONAL - **Target Enrollment:** 500 - **Lead Sponsor:** Sarepta Therapeutics, Inc. - **Conditions:** Duchenne Muscular Dystrophy - **Start Date:** 2024-02-07 - **Completion Date:** 2038-12-31 - **CT.gov Last Update:** 2026-05-04 ## Brief Summary 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. ## Eligibility - **Minimum age:** 4 Years - **Sex:** MALE - **Healthy Volunteers:** No ``` Inclusion Criteria: * Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. * Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c): * Is at least 4 years of age at the time of infusion * Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval For Standard of Care Comparators (Cohort 2): * Is at least 4 years of age at the time of enrollment * Is unexposed to DMD gene therapy at the time of this observational study enrollment 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: * The participant's ability to comply with the protocol-required procedures, * The participant's wellbeing or safety, and/or * The clinical interpretability of the data collected from the participant. Other inclusion/exclusion criteria may apply. ``` ## Arms - **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. ## Interventions - **Delandistrogene Moxeparvovec** (GENETIC) — No study medication will be provided by the sponsor during this study. - **Standard of Care** (DRUG) — No study medication will be provided by the sponsor during this study. ## Primary Outcomes - **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)_ ## Secondary Outcomes - **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)_ ## Locations (25) - 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 - 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 - University of Kansas Medical Center, Kansas City, Kansas, United States - University of Michigan, Ann Arbor, Michigan, United States - Duke Lenox Baker Children's Hospital, Durham, North Carolina, United States - Childrens Hospital Medical Center of Akron, Akron, Ohio, United States - OHSU Healthcare (Oregon Health and Science University), Portland, Oregon, United States - Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania, United States - Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States - Le Bonheur Children's Hospital, Memphis, Tennessee, United States - Vanderbilt University Medical Center, Nashville, Tennessee, United States - Cook Children's Hospital, Fort Worth, Texas, United States - University of Virginia, Charlottesville, Virginia, United States - Children's Hospital of the King's Daughters, Norfolk, Virginia, United States - Seattle Children's Hospital, Seattle, Washington, United States - The Board of Regents of the University of Wisconsin, Madison, Wisconsin, United States - The Medical College of Wisconsin, Milwaukee, Wisconsin, United States ## Recent Field Changes (last 30 days) - `locations.university of michigan|ann arbor|michigan|united states` — added _(2026-05-12)_ - `locations.university of colorado - ppds|aurora|colorado|united states` — added _(2026-05-12)_ - `locations.connecticut children's medical center - hartford|hartford|connecticut|united states` — added _(2026-05-12)_ - `locations.children's national medical center|washington d.c.|district of columbia|united states` — added _(2026-05-12)_ - `locations.nicklaus children's hospital|miami|florida|united states` — added _(2026-05-12)_ - `locations.nemours children's hospital - orlando|orlando|florida|united states` — added _(2026-05-12)_ - `locations.all children's research institute, inc|st. petersburg|florida|united states` — added _(2026-05-12)_ - `locations.ann and robert h lurie childrens hospital of chicago|chicago|illinois|united states` — added _(2026-05-12)_ - `status.overallStatus` — added _(2026-05-12)_ - `status.primaryCompletionDate` — added _(2026-05-12)_ - `status.completionDate` — added _(2026-05-12)_ - `status.lastUpdatePostDate` — added _(2026-05-12)_ - `design.enrollmentCount` — added _(2026-05-12)_ - `locations.indiana clinical and translational science institute|indianapolis|indiana|united states` — added _(2026-05-12)_ - `locations.university of kansas medical center|kansas city|kansas|united states` — added _(2026-05-12)_ - `eligibility.criteria` — added _(2026-05-12)_ - `eligibility.minAge` — added _(2026-05-12)_ - `eligibility.sex` — added _(2026-05-12)_ - `outcomes.primary` — added _(2026-05-12)_ - `outcomes.secondary` — added _(2026-05-12)_ - `armsInterventions.arms` — added _(2026-05-12)_ - `armsInterventions.interventions` — added _(2026-05-12)_ - `sponsor.lead` — added _(2026-05-12)_ - `results.hasResults` — added _(2026-05-12)_ - `locations.arkansas children's hospital|little rock|arkansas|united states` — added _(2026-05-12)_ - `locations.children's hospital los angeles - pin|los angeles|california|united states` — added _(2026-05-12)_ - `locations.duke lenox baker children's hospital|durham|north carolina|united states` — added _(2026-05-12)_ - `locations.childrens hospital medical center of akron|akron|ohio|united states` — added _(2026-05-12)_ - `locations.ohsu healthcare (oregon health and science university)|portland|oregon|united states` — added _(2026-05-12)_ - `locations.penn state health milton s. hershey medical center|hershey|pennsylvania|united states` — added _(2026-05-12)_ - `locations.children's hospital of philadelphia|philadelphia|pennsylvania|united states` — added _(2026-05-12)_ - `locations.le bonheur children's hospital|memphis|tennessee|united states` — added _(2026-05-12)_ - `locations.vanderbilt university medical center|nashville|tennessee|united states` — added _(2026-05-12)_ - `locations.cook children's hospital|fort worth|texas|united states` — added _(2026-05-12)_ - `locations.university of virginia|charlottesville|virginia|united states` — added _(2026-05-12)_ - `locations.children's hospital of the king's daughters|norfolk|virginia|united states` — added _(2026-05-12)_ - `locations.seattle children's hospital|seattle|washington|united states` — added _(2026-05-12)_ - `locations.the board of regents of the university of wisconsin|madison|wisconsin|united states` — added _(2026-05-12)_ - `locations.the medical college of wisconsin|milwaukee|wisconsin|united states` — added _(2026-05-12)_ --- *Canonical: https://parkinsonspathways.com/agent/trials/NCT06270719.md* *Source data (authoritative): https://clinicaltrials.gov/study/NCT06270719* *This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*