Clinical trial NCT06606340: A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Official title: A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
DMD
Post-marketing
Observational
Phase 4
Eteplirsen
Golodirsen
Casimersen
Lead sponsor: Sarepta Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2024-09-23
Enrollment: 300
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants within each prescriber's practice who are receiving eteplirsen, golodirsen, or casimersen, in routine clinical practice, and who meet the study eligibility criteria and provide informed consent (either by the participant or through authorization by a legal guardian), will be invited to enroll into the study and will be followed according to the protocol.

Brief 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 criteria: 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 outcomes:
measure: Loss of Ambulation (LOA)
timeFrame: Up to 5 years
measure: Time to Rise From the Floor (Supine to Stand)
timeFrame: Up to 5 years
measure: Time to Walk/Run 10 Meters
timeFrame: Up to 5 years
measure: Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A
timeFrame: Up to 5 years
measure: Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)
timeFrame: Up to 5 years
measure: Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)
timeFrame: Up to 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital Research Institute
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: Washington
country: United States
facility: Children's National Hospital
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Iowa City
country: United States
facility: University Of Iowa Hospitals And Clinics
state: Iowa
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: New York
country: United States
facility: Columbia University Medical Center - PIN
state: New York
city: Durham
country: United States
facility: Duke Lenox Baker Children's Hospital
state: North Carolina
city: Winston-Salem
country: United States
facility: Atrium Health Wake Forest University Health Sciences
state: North Carolina
city: Cincinnati
country: United States
facility: Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Hershey
country: United States
facility: Penn State Health Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Fort Worth
country: United States
facility: Cook Children's Hospital
state: Texas
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Charlottesville
country: United States
facility: UVA Children's Hospital
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
References: