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

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]

Observational Catalyst Pharmaceuticals, Inc. · NCT06564974

This study is looking at how safe the drug Vamorolone is for boys with Duchenne Muscular Dystrophy and how it affects their quality of life over five years.

Quick facts

Study typeObservational
Enrollment250 (estimated)
Ages2 Years and up
SexMale
SponsorCatalyst Pharmaceuticals, Inc. Industry-sponsored
Locations27 sites (Phoenix, Arizona and 26 other locations)
Trial IDNCT06564974 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates for this study are male patients aged 2 years and older who have a confirmed diagnosis of Duchenne Muscular Dystrophy and are currently being treated with AGAMREE®.

Not a fit: Patients who have contraindications to AGAMREE® or other medical conditions that could affect their participation may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety and effectiveness of Vamorolone for patients with Duchenne Muscular Dystrophy.

How similar studies have performed: While this study is observational, similar approaches in monitoring long-term safety and quality of life in DMD patients have shown promise, indicating potential for valuable findings.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 this trial is running

Phoenix, Arizona and 26 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Duchenne Muscular DystrophyNeuromuscularAGAMREE®VamoroloneDMDMuscular Dystrophy
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.