NCT06564974From ClinicalTrials.govRecruiting

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

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

Duchenne Muscular Dystrophy

At a glance

Phase: Phase not stated
Study type: Observational
Sponsor: Catalyst Pharmaceuticals, Inc.
Enrolment target: 250
Started: 25 September 2024
Main results due: February 2032
Study sites: 33
Registry updated: 24 September 2026

Can you take part?

Aged 2 years and over.
Men only.
You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full 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.

What this study is about

In the sponsor’s own words, from the registry.

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).

Study sites(33)

Phoenix Children's Hospital
Phoenix, Arizona, United States
Recruiting
Arkansas Childrens Hospital
Little Rock, Arkansas, United States
Enrolling by invitation
Loma Linda University Pediatric Subspecialty Clinics
Loma Linda, California, United States
Recruiting
Children's Hospital Los Angeles
Los Angeles, California, United States
Not yet recruiting
Valley Children's Hospital
Madera, California, United States
Not yet recruiting
Stanford University
Palo Alto, California, United States
Not yet recruiting
University of California, Davis
Sacramento, California, United States
Recruiting
Children's National Health System
Washington D.C., District of Columbia, United States
Not yet recruiting
University of Florida Clinical and Translational Science Institue
Gainesville, Florida, United States
Recruiting
University of Miami
Miami, Florida, United States
Not yet recruiting
Nicklaus Children's Hospital
Miami, Florida, United States
Enrolling by invitation
Nemours Children's Hospital
Orlando, Florida, United States
Enrolling by invitation
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States
Recruiting
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Enrolling by invitation
Indiana University Health - Riley Hospital for Children
Indianapolis, Indiana, United States
Enrolling by invitation
University of Kansas Medical Center
Kansas City, Kansas, United States
Enrolling by invitation
University of Massachusetts Memorial Medical Center
North Worcester, Massachusetts, United States
Enrolling by invitation
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Enrolling by invitation
Masonic Children's Hospital - Discovery Clinic
Minneapolis, Minnesota, United States
Not yet recruiting
The Children's Hospital at Montefiore
The Bronx, New York, United States
Not yet recruiting
Atrium Health Neurosciences Institute
Charlotte, North Carolina, United States
Enrolling by invitation
Duke University Medical Center and Childrens Health Center
Durham, North Carolina, United States
Enrolling by invitation
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Recruiting
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
Hershey, Pennsylvania, United States
Enrolling by invitation
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Enrolling by invitation
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Enrolling by invitation
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
Denton, Texas, United States
Enrolling by invitation
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Recruiting
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
Charlottesville, Virginia, United States
Enrolling by invitation
Seattle Children's Hospital
Seattle, Washington, United States
Enrolling by invitation
Children's Wisconsin
Milwaukee, Wisconsin, United States
Not yet recruiting
San Jorge Children's Hospital
San Juan, Puerto Rico, Puerto Rico
Recruiting
FDI Clinical Research
San Juan, Puerto Rico, Puerto Rico
Enrolling by invitation

Showing 33 of 33 sites. 8 of the 33 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT06564974. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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