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)]
Catalyst Pharmaceuticals, Inc.
250 participants
Sep 25, 2024
OBSERVATIONAL
Conditions
Summary
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).
Eligibility
Inclusion Criteria5
- 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 Criteria1
- \. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.
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Interventions
Study treatment is AGAMREE®, which is commercially available as an oral suspension.
Locations(27)
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NCT06564974