Observing long-term safety of Vamorolone in boys with Duchenne Muscular Dystrophy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational registry study aims to monitor the long-term safety and quality of life of male patients aged 2 years and older who are being treated with the FDA-approved drug AGAMREE® for Duchenne Muscular Dystrophy (DMD). Conducted across approximately 25 sites in the US, the study plans to enroll around 250 participants and will follow them for about 5 years. Evaluations will include various health parameters such as growth, physical performance, cardiovascular status, and quality of life, with yearly follow-up visits to collect comprehensive data.

Why it matters

Who can join: the full rules

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.

Where it's running

• Phoenix Children's Hospital
  Phoenix, Arizona, United States Recruiting
• Loma Linda University Pediatric Subspecialty Clinics
  Loma Linda, California, United States Recruiting
• University of California, Davis
  Sacramento, California, United States Recruiting
• University of Florida Clinical and Translational Science Institue
  Gainesville, Florida, United States Recruiting
• Johns Hopkins All Children's Hospital
  St. Petersburg, Florida, United States Recruiting
• Cincinnati Children's Hospital Medical Center
  Cincinnati, Ohio, United States Recruiting

Study contacts

• Principal investigator: Aravindham Veerapandiyan, MD — Arkansas Childrens Hospital
• Study coordinator: Mark Wingertzahn, PhD
• Email: mark.wingertzahn@catalystpharma.com
• Phone: (305) 420-3200

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.