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

Recruitingn/aAges 2 Years - anyMaleCatalyst Pharmaceuticals, Inc.
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Inclusion Criteria: 1. 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. 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 anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot). 5. Patient has a current, active prescription for, or is on, 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.
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What it tests

  • Vamorolone
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Where

33 sites
Phoenix Children's Hospital
Phoenix, United States
Arkansas Childrens Hospital
Little Rock, United States
Loma Linda University Pediatric Subspecialty Clinics
Loma Linda, United States
Children's Hospital Los Angeles
Los Angeles, United States
Valley Children's Hospital
Madera, United States
Stanford University
Palo Alto, United States
University of California, Davis
Sacramento, United States
Children's National Health System
Washington D.C., United States

…and 25 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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