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

Launched by CATALYST PHARMACEUTICALS, INC. · Aug 19, 2024

Keywords

ClinConnect Summary

This clinical trial is studying the long-term safety and quality of life for boys aged 2 years and older who have Duchenne Muscular Dystrophy (DMD) and are being treated with a medication called AGAMREE® (Vamorolone). The researchers want to see how well the drug works over time and how it affects the daily lives of these patients. This study is currently recruiting participants, which means they are looking for boys who meet certain criteria to join.

To be eligible for the study, boys must be at least 2 years old, have a confirmed diagnosis of DMD, and be currently taking AGAMREE®. They also need to have their parent or guardian willing to consent to the study and complete some quality of life questionnaires. Participants will be closely monitored throughout the study, and their experiences will help researchers understand the long-term effects of AGAMREE®. If you or someone you know might be interested, it’s a good idea to talk to a healthcare provider for more information.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 1. Patient or parent/guardian willing and able to provide written informed consent after the nature of the registry has been explained and before the start of any registry-related procedures.
• • 2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
• • 3. Male patients at least 2 years old.
• • 4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to antidystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
• • 5. Currently on treatment with AGAMREE®.
• Exclusion Criteria:
• • 1. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the investigator, would affect registry participation, performance, or interpretation of registry assessments.