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NCT06564974 · ClinicalTrials.gov registry record
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
A clinical trial of Duchenne Muscular Dystrophy, sponsored by Catalyst Pharmaceuticals.
- Recruiting
- Registry status
- 250
- Enrollment target
- 20
- Study locations
NCT06564974: Recruiting study of Duchenne Muscular Dystrophy, sponsored by Catalyst Pharmaceuticals.
NCT06564974 is a study of Duchenne Muscular Dystrophy that is actively recruiting participants, run by Catalyst Pharmaceuticals. The registered enrollment target is 250 participants, below the 407-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (39% lower). The trial reports 20 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06564974, a study of Duchenne Muscular Dystrophy, is actively recruiting participants, sponsored by Catalyst Pharmaceuticals.
- RECRUITING
- Registry status
- 250 participants
- Enrollment target
- 20
- Study locations
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).
Primary Outcome
Standing height in participants 2 years of age and older will be measured using a stadiometer mounted at a right angle between a level floor and against a straight, vertical surface. When transitioning from recumbent length to standing height measurements in participants between 2 to 3 years of age, measure both length and height. If collecting standing height measurements is not feasible, height will be estimated using arm span measurements. To estimate the height, the arm span will be measured
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 |
What NCT06564974 shows while recruiting
NCT06564974 is an observational study that tracks outcomes without assigning an intervention. The registered 250 participants enrollment target is mid-sized for trials with a published cap, below the 407-participant average among 48 other Duchenne Muscular Dystrophy trials with a reported enrollment target (39% lower).
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.
NCT06564974 names 20 study sites across 13 states, led by Florida, California, North Carolina.
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.
What interventions are being tested in trial NCT06564974?
The interventions under investigation include: Vamorolone (DRUG).
Who is sponsoring clinical trial NCT06564974?
This trial is sponsored by Catalyst Pharmaceuticals, which has 10 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Duchenne Muscular Dystrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT06564974's enrollment target sits among peer trials
250 6th of 48 higher than 42 of 48 other Duchenne Muscular Dystrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Duchenne Muscular Dystrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
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The Duchenne Registry
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