Status:
RECRUITING
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.
Lead Sponsor:
Catalyst Pharmaceuticals, Inc.
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
2+ years
Brief Summary
The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term s...
Detailed Description
This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®. This registry will be conduc...
Eligibility Criteria
Inclusion
- 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.
- 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 antidystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
- Currently on treatment with AGAMREE®.
Exclusion
- 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.
Key Trial Info
Start Date :
July 31 2024
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
November 1 2030
Estimated Enrollment :
250 Patients enrolled
Trial Details
Trial ID
NCT06564974
Start Date
July 31 2024
End Date
November 1 2030
Last Update
August 5 2025
Active Locations (25)
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1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
2
Arkansas Childrens Hospital
Little Rock, Arkansas, United States, 72202
3
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
4
Stanford University
Palo Alto, California, United States, 94305