Long-term safety of vamorolone in boys with Duchenne muscular dystrophy
Observes long-term safety and quality of life in boys with Duchenne muscular dystrophy taking vamorolone.
Plain-language title generated from the registry record
Registry title: Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Key facts
- Where
- Charlotte, Charlottesville, Chicago, Cincinnati, Dallas, Denton, Durham, Gainesville, Grand Rapids, Hershey, Indianapolis, Kansas City, Little Rock, Loma Linda, Los Angeles, Madera, Miami, Milwaukee, Minneapolis, North Worcester, Orlando, Palo Alto, Philadelphia, Phoenix, Sacramento, San Antonio, Seattle, St. Petersburg, The Bronx, and Washington D.C., United StatesSee the 31 sites
Where in United States
- Atrium Health Neurosciences InstituteCharlotte, United StatesEnrolling by invitation
- University of Virginia Health System (UVAHS) - Pediatric Neuromuscular CenterCharlottesville, United StatesEnrolling by invitation
- Lurie Children's Hospital of ChicagoChicago, United StatesEnrolling by invitation
- Cincinnati Children's Hospital Medical CenterCincinnati, United StatesTaking people
- University of Texas Southwestern Medical CenterDallas, United StatesEnrolling by invitation
- Neurology Rare Disease Center - Neurology & Neuromuscular Care CenterDenton, United StatesEnrolling by invitation
- Duke University Medical Center and Childrens Health CenterDurham, United StatesEnrolling by invitation
- University of Florida Clinical and Translational Science InstitueGainesville, United StatesTaking people
- Helen DeVos Children's HospitalGrand Rapids, United StatesEnrolling by invitation
- Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience InstituteHershey, United StatesEnrolling by invitation
- Indiana University Health Riley Hospital for ChildrenIndianapolis, United StatesEnrolling by invitation
- University of Kansas Medical CenterKansas City, United StatesEnrolling by invitation
- Arkansas Childrens HospitalLittle Rock, United StatesEnrolling by invitation
- Loma Linda University Pediatric Subspecialty ClinicsLoma Linda, United StatesTaking people
- Children's Hospital Los AngelesLos Angeles, United StatesNot yet recruiting
- Valley Children's HospitalMadera, United StatesNot yet recruiting
- Nicklaus Children's HospitalMiami, United StatesEnrolling by invitation
- University of MiamiMiami, United StatesNot yet recruiting
- Children's WisconsinMilwaukee, United StatesNot yet recruiting
- Masonic Children's Hospital - Discovery ClinicMinneapolis, United StatesNot yet recruiting
- University of Massachusetts Memorial Medical CenterNorth Worcester, United StatesEnrolling by invitation
- Nemours Children's HospitalOrlando, United StatesEnrolling by invitation
- Stanford UniversityPalo Alto, United StatesNot yet recruiting
- Children's Hospital of Philadelphia (CHOP)Philadelphia, United StatesEnrolling by invitation
- Phoenix Children s HospitalPhoenix, United StatesTaking people
- University of California - DavisSacramento, United StatesTaking people
- The University of Texas Health Science Center at San AntonioSan Antonio, United StatesTaking people
- Seattle Children's HospitalSeattle, United StatesEnrolling by invitation
- Johns Hopkins All Children's HospitalSt. Petersburg, United StatesTaking people
- The Children's Hospital at MontefioreThe Bronx, United StatesNot yet recruiting
- Children's National Health SystemWashington D.C., United StatesNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Catalyst Pharmaceuticals, Inc.
- Trial code
- NCT06564974
- Record updated
- 24 September 2026
- Original description
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).Read full original descriptionShow less
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]
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).
Inclusion: 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; 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 anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot); Patient has a current, active prescription for, or is on, AGAMREE®.
Exclusion: Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.