A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

NCT06606340 · tracked via the Priya Life Science USA tracker

Sponsor

Sarepta Therapeutics, Inc.

Phase

Observational

Started

2019-01-07

Last updated

2025-09-10

Condition(s) studied

Duchenne Muscular Dystrophy

Investigational drug(s) / intervention(s)

EteplirsenGolodirsenCasimersen

Eteplirsen: No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.

Golodirsen: No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.

Casimersen: No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.

Study summary

This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.

Eligibility

Sex

MALE

Min age

—

Max age

—

Healthy volunteers

Key Inclusion Criteria: * Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures. * Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report. * Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled. Key Exclusion Criteria: * Is currently participating in any DMD interventional study at the time of this study enrollment. * Has declined to provide consent for collection of their genetic data. * Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise: 1. The participant's ability to comply with the protocol-required procedures 2. The participant's wellbeing or safety, and/or 3. The clinical interpretability of the data collected from the participant. Other inclusion/exclusion criteria may apply.

Primary outcome measure(s)

Loss of Ambulation (LOA) — Up to 5 years
Time to Rise From the Floor (Supine to Stand) — Up to 5 years
Time to Walk/Run 10 Meters — Up to 5 years
Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A — Up to 5 years
Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted) — Up to 5 years
Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO) — Up to 5 years

Trial sites (20)

Facility	City	Region
Arkansas Children's Hospital Research Institute	Little Rock	Arkansas
Children's Hospital Los Angeles	Los Angeles	California
University of California Davis Medical Center	Sacramento	California
Children's National Hospital	Washington D.C.	District of Columbia
University of Florida	Gainesville	Florida
University Of Iowa Hospitals And Clinics	Iowa City	Iowa
Kennedy Krieger Institute	Baltimore	Maryland
Washington University	St Louis	Missouri
Columbia University Medical Center - PIN	New York	New York
Duke Lenox Baker Children's Hospital	Durham	North Carolina
Atrium Health Wake Forest University Health Sciences	Winston-Salem	North Carolina
Children's Hospital Medical Center	Cincinnati	Ohio
Nationwide Children's Hospital	Columbus	Ohio
Penn State Health Milton S. Hershey Medical Center	Hershey	Pennsylvania
Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
Cook Children's Hospital	Fort Worth	Texas
Texas Children's Hospital	Houston	Texas
UVA Children's Hospital	Charlottesville	Virginia
Seattle Children's Hospital	Seattle	Washington

More Sarepta Therapeutics, Inc. trials in the USA

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) Active, not recruiting

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 Active, not recruiting

A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency) Active, not recruiting

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice Active, not recruiting

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) Active, not recruiting

Other trials for the same condition

A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX) Active, not recruiting

Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53) Recruiting

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) Recruiting

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study Recruiting

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06606340 on ClinicalTrials.gov ↗ ← All trials in the USA