DMD-001 SUMMIT Registry Study: Long-Term Safety and Quality of Life With AGAMREE in Duchenne Muscular Dystrophy
Actively RecruitingObservationalReducing Inflammation
Study Overview
- Age
- 2 years and older
- Phase
- Observational
- Sponsor
- Catalyst Pharmaceuticals
- Therapeutic Approach
- Reducing Inflammation
- Variant Requirement
- Patient has a current, active prescription for, or is on, AGAMREE.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2024-09-25
- Primary Completion (Estimated)
- 2032-02
- Study Completion (Estimated)
- 2032-02
- Enrollment (Estimated)
- 250
- Countries
- United StatesPuerto Rico
Study Requirements and Criteria
Inclusion Criteria
- 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).
Exclusion Criteria
Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.
Contact Information
This section provides contact details for people who can answer questions about joining this study
- Name: Syune Nersisyan, PhD
- Phone Number: (305) 420-3200
- Email: [email protected]
Clinical Trial Registry
NCT ID
NCT06564974This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.