NCT06564974 Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
| NCT ID | NCT06564974 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Catalyst Pharmaceuticals, Inc. |
| Condition | Duchenne Muscular Dystrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 250 participants |
| Start Date | 2024-09-25 |
| Primary Completion | 2032-02 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 250 participants in total. It began in 2024-09-25 with a primary completion date of 2032-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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).
Eligibility 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.
Contact & Investigator
William Andrews, MD
STUDY DIRECTOR
Catalyst Pharmaceuticals
Frequently Asked Questions
Who can join the NCT06564974 clinical trial?
This trial is open to male participants only, aged 2 Years or older, studying Duchenne Muscular Dystrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06564974 currently recruiting?
Yes, NCT06564974 is actively recruiting participants. Contact the research team at mark.wingertzahn@catalystpharma.com for enrollment information.
Where is the NCT06564974 trial being conducted?
This trial is being conducted at Phoenix, United States, Little Rock, United States, Loma Linda, United States, Los Angeles, United States and 11 additional locations.
Who is sponsoring the NCT06564974 clinical trial?
NCT06564974 is sponsored by Catalyst Pharmaceuticals, Inc.. The principal investigator is William Andrews, MD at Catalyst Pharmaceuticals. The trial plans to enroll 250 participants.