RECRUITINGOBSERVATIONAL
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]
About This Trial
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).
Who May Be Eligible (Plain English)
Who May Qualify:
1. Patient or parent/legal guardian is willing and able to provide written willing to sign a consent form once the nature of the registry has been explained and prior to the start of any registry-related procedures.
2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
3. Male patients at least 2 years old.
4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
5. Patient has a current, active prescription for, or is on, AGAMREE®.
Who Should NOT Join This Trial:
1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
1. Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
3. Male patients at least 2 years old.
4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
5. Patient has a current, active prescription for, or is on, AGAMREE®.
Exclusion Criteria:
1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.
Treatments Being Tested
DRUG
Vamorolone
Study treatment is AGAMREE®, which is commercially available as an oral suspension.
Locations (20)
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