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

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
Sarepta Therapeutics, Inc.
Study ID
NCT06606340
Status
Enrolling By Invitation

Conditions

Eligibility Criteria

Sex
MALE
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Eteplirsen — DRUG
    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 — DRUG
    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 — DRUG
    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 Details

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.

Key Dates

Start date
Jan 7, 2019
Status verified
Aug 2025
Primary completion
Dec 31, 2033
Completion
Dec 31, 2033

Study Design

Enrollment
300 participants (estimated)

Arms

  • Arm: Eteplirsen
  • Arm: Golodirsen
  • Arm: Casimersen

Primary Outcome Measure

Loss of Ambulation (LOA) [ Time Frame: Up to 5 years ]

Locations (20)

FacilityCityStateZIPSite coordinators
Arkansas Children's Hospital Research InstituteLittle RockArkansas72202-
Children's Hospital Los AngelesLos AngelesCalifornia90027-
University of California Davis Medical CenterSacramentoCalifornia95817-
Children's National HospitalWashington D.C.District of Columbia20010-
University of FloridaGainesvilleFlorida32610-
University Of Iowa Hospitals And ClinicsIowa CityIowa52242-
Kennedy Krieger InstituteBaltimoreMaryland21287-
Washington UniversitySt LouisMissouri63110-
Columbia University Medical Center - PINNew YorkNew York10032-
Duke Lenox Baker Children's HospitalDurhamNorth Carolina27705-
Atrium Health Wake Forest University Health SciencesWinston-SalemNorth Carolina27157-
Children's Hospital Medical CenterCincinnatiOhio45229-
Nationwide Children's HospitalColumbusOhio43205-
Penn State Health Milton S. Hershey Medical CenterHersheyPennsylvania17033-0850-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
UPMC Children's Hospital of PittsburghPittsburghPennsylvania15213-
Cook Children's HospitalFort WorthTexas76104-
Texas Children's HospitalHoustonTexas77030-
UVA Children's HospitalCharlottesvilleVirginia22908-
Seattle Children's HospitalSeattleWashington98105-

Find similar trials in Little Rock, AR

By condition
Duchenne muscular dystrophy
By specialty
Pediatrics
By research site
Arkansas Children's Hospital Research Institute· Little Rock, ARChildren's Hospital Los Angeles· Los Angeles, CAUniversity of California Davis Medical Center· Sacramento, CAChildren's National Hospital· Washington D.C., DCUniversity of Florida· Gainesville, FLUniversity Of Iowa Hospitals And Clinics· Iowa City, IA

Related Studies