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Study identifier: NCT06564974 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Condition: Duchenne Muscular Dystrophy  ·  Sponsor: Catalyst Pharmaceuticals, Inc.

PhaseN/A
Planned participants250
Who can joinMale, 2 Years to no upper limit
Healthy volunteersNo

About this study

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).

This description comes directly from the study's public registry record.

Talk to the study team

Mark Wingertzahn, PhD  ·  (305) 420-3200  ·  mark.wingertzahn@catalystpharma.com

Always discuss trial participation with your own doctor first.

Locations (33)

Phoenix Children's HospitalPhoenix, Arizona, United StatesRecruiting
Arkansas Childrens HospitalLittle Rock, Arkansas, United StatesRecruiting
Loma Linda University Pediatric Subspecialty ClinicsLoma Linda, California, United StatesNot Yet Recruiting
Children's Hospital Los AngelesLos Angeles, California, United StatesNot Yet Recruiting
Valley Children's HospitalMadera, California, United StatesNot Yet Recruiting
Stanford UniversityPalo Alto, California, United StatesNot Yet Recruiting
University of California, DavisSacramento, California, United StatesNot Yet Recruiting
Children's National Health SystemWashington D.C., District of Columbia, United StatesNot Yet Recruiting
University of Florida Clinical and Translational Science InstitueGainesville, Florida, United StatesNot Yet Recruiting
University of MiamiMiami, Florida, United StatesNot Yet Recruiting
Nicklaus Children's HospitalMiami, Florida, United StatesRecruiting
Nemours Children's HospitalOrlando, Florida, United StatesRecruiting

+ 21 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT06564974