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Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Sponsor: Catalyst Pharmaceuticals, Inc.
Summary
The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).
Official title: Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]
Key Details
Gender
MALE
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
250
Start Date
2024-09-25
Completion Date
2032-02
Last Updated
2026-02-27
Healthy Volunteers
No
Conditions
Interventions
Vamorolone
Study treatment is AGAMREE®, which is commercially available as an oral suspension.
Locations (27)
Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Childrens Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of California, Davis
Sacramento, California, United States
University of Florida Clinical and Translational Science Institue
Gainesville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Indiana University Health - Riley Hospital for Children
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Massachusetts Memorial Medical Center
North Worcester, Massachusetts, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Atrium Health Neurosciences Institute
Charlotte, North Carolina, United States
Duke University Medical Center and Childrens Health Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
Denton, Texas, United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
Charlottesville, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
Children's Wisconsin
Milwaukee, Wisconsin, United States
San Jorge Children's Hospital
San Juan, Puerto Rico, Puerto Rico
FDI Clinical Research
San Juan, Puerto Rico, Puerto Rico