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Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
NCT06564974 · View on ClinicalTrials.gov ↗
Study 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).
Conditions Studied
Interventions
- DRUG Vamorolone
Study Locations (20)
Florida
- University of Florida Clinical and Translational Science Institue — Gainesville
- Nicklaus Children's Hospital — Miami
- Nemours Children's Hospital — Orlando
- Johns Hopkins All Children's Hospital — St. Petersburg
California
- Children's Hospital Los Angeles — Los Angeles
- Stanford University — Palo Alto
- University of California, Davis — Sacramento
North Carolina
- Atrium Health Neurosciences Institute — Charlotte
- Duke University Medical Center and Childrens Health Center — Durham
Pennsylvania
- Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute — Hershey
- Children's Hospital of Philadelphia (CHOP) — Philadelphia
Arizona
- Phoenix Children's Hospital — Phoenix
Arkansas
- Arkansas Childrens Hospital — Little Rock
Illinois
- Lurie Children's Hospital of Chicago — Chicago
Indiana
- Indiana University Health - Riley Hospital for Children — Indianapolis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 250 participants |
| Start Date | 2024-09-25 |
| Est. Completion | 2032-02 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06564974
The ClinicalTrials.gov registry entry for NCT06564974 describes a study currently listed as recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 250 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Catalyst Pharmaceuticals, which has 6 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Duchenne Muscular Dystrophy appearing as the primary indexed condition, and to 1 intervention — of which Vamorolone is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06564974 reports 20 study locations spanning 13 distinct geographic areas — top geographies include Florida, California, North Carolina. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06564974 about?
NCT06564974 is a clinical study titled "Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT". 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...
What is the current status of trial NCT06564974?
This trial is currently recruiting. The enrollment target is 250 participants. The study started on 2024-09-25. Estimated completion is 2032-02.
What conditions does trial NCT06564974 study?
This clinical trial studies the following conditions: Duchenne Muscular Dystrophy. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06564974?
The interventions under investigation include: Vamorolone (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06564974?
This trial is sponsored by Catalyst Pharmaceuticals, which has 6 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06564974 being conducted?
This trial has 20 study locations across Arizona, Arkansas, California, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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