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).
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]
This is a multi-center, observational, prospective, longitudinal registry study, designed to collect safety data 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 DMD.
Primary Objective is to collect additional information on the safety of long-term treatment with AGAMREE® in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD) as determined by:
Growth parameters Body mass index (BMI) Musculoskeletal assessments Ophthalmological assessments Cardiovascular assessments Pubertal development assessments Adverse events (AEs)
Secondary Objective is to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires).
This registry will be conducted in the US, at approximately 40 sites known to treat and follow patients with DMD. The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD.
Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 60 days) for assessments. Information on some historical and ongoing standard of care treatment and procedures for management of DMD will also be collected.
