Status: Recruiting
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-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).
Participants needed: 250
Trial details
Age: 2+Biological sex: MaleType: ObservationalSponsor: Catalyst Pharmaceuticals, Inc.Updated: Feb 27, 2026Locations: 27Duration: 5 Years
Eligibility criteria
Patient or parent/legal guardian is willing and able to provide written informed... [+4]