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

Enrolling by invitationOBSERVATIONAL
Enrollment

300

Participants

Timeline

Start Date

January 7, 2019

Primary Completion Date

December 31, 2033

Study Completion Date

December 31, 2033

Conditions
Duchenne Muscular Dystrophy
Interventions
DRUG

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.

DRUG

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.

DRUG

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.

Trial Locations (20)

10032

Columbia University Medical Center - PIN, New York

15213

UPMC Children's Hospital of Pittsburgh, Pittsburgh

19104

Children's Hospital of Philadelphia, Philadelphia

20010

Children's National Hospital, Washington D.C.

21287

Kennedy Krieger Institute, Baltimore

22908

UVA Children's Hospital, Charlottesville

27157

Atrium Health Wake Forest University Health Sciences, Winston-Salem

27705

Duke Lenox Baker Children's Hospital, Durham

32610

University of Florida, Gainesville

43205

Nationwide Children's Hospital, Columbus

45229

Children's Hospital Medical Center, Cincinnati

52242

University Of Iowa Hospitals And Clinics, Iowa City

63110

Washington University, St Louis

72202

Arkansas Children's Hospital Research Institute, Little Rock

76104

Cook Children's Hospital, Fort Worth

77030

Texas Children's Hospital, Houston

90027

Children's Hospital Los Angeles, Los Angeles

95817

University of California Davis Medical Center, Sacramento

98105

Seattle Children's Hospital, Seattle

17033-0850

Penn State Health Milton S. Hershey Medical Center, Hershey

All Listed Sponsors
lead

Sarepta Therapeutics, Inc.

INDUSTRY