Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy
Status: Recruiting
Location: See all (25) locations...
Intervention Type: Drug
Study Type: Observational
SUMMARY
The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term safety and quality of life.
Eligibility
Participation Requirements
Sex: Male
Minimum Age: 2
Healthy Volunteers: f
View:
• Patient or parent/guardian willing and able to provide written informed consent after the nature of the registry has been explained and before the start of any registry-related procedures.
• Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
• Male patients at least 2 years old.
• Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to antidystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
• Currently on treatment with AGAMREE®.
Locations
United States
Arkansas
Arkansas Childrens Hospital
RECRUITING
Little Rock
Arizona
Phoenix Children's Hospital
RECRUITING
Phoenix
California
Children's Hospital Los Angeles
NOT_YET_RECRUITING
Los Angeles
Stanford University
NOT_YET_RECRUITING
Palo Alto
University of California, Davis
NOT_YET_RECRUITING
Sacramento
Florida
University of Florida Clinical and Translational Science Institue
NOT_YET_RECRUITING
Gainesville
Nicklaus Children's Hospital
RECRUITING
Miami
Nemours Children's Hospital
RECRUITING
Orlando
Illinois
Lurie Children's Hospital of Chicago
RECRUITING
Chicago
Indiana
Indiana University Health - Riley Hospital for Children
NOT_YET_RECRUITING
Indianapolis
Kansas
University of Kansas Medical Center
RECRUITING
Kansas City
Massachusetts
University of Massachusetts Memorial Medical Center
RECRUITING
North Worcester
Michigan
Helen DeVos Children's Hospital
RECRUITING
Grand Rapids
North Carolina
Atrium Health Neurosciences Institute
RECRUITING
Charlotte
Duke University Medical Center and Childrens Health Center
RECRUITING
Durham
Ohio
Cincinnati Children's Hospital Medical Center
RECRUITING
Cincinnati
Pennsylvania
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
RECRUITING
Hershey
Children's Hospital of Philadelphia (CHOP)
RECRUITING
Philadelphia
Texas
University of Texas Southwestern Medical Center
RECRUITING
Dallas
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
RECRUITING
Denton
The University of Texas Health Science Center at San Antonio
RECRUITING
San Antonio
Virginia
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center