Actively Recruiting
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Led by Catalyst Pharmaceuticals, Inc. · Updated on 2026-02-27
250
Participants Needed
27
Research Sites
383 weeks
Total Duration
On this page
Sponsors
C
Catalyst Pharmaceuticals, Inc.
Lead Sponsor
I
ICON plc
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
Official Title
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient or parent/legal guardian is willing and able to provide written informed consent before any registry procedures.
- Patient and/or parent/guardian are willing and able to complete quality of life questionnaires.
- Male patients aged 2 years or older.
- Confirmed diagnosis of Duchenne muscular dystrophy by genetic testing or muscle biopsy.
- Patient currently has an active prescription for or is taking AGAMREE® (Vamorolone).
You will not qualify if you...
- Any contraindication to AGAMREE® or medical condition that could affect participation or assessment results, as judged by the Investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 27 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Arkansas Childrens Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Not Yet Recruiting
4
Stanford University
Palo Alto, California, United States, 94305
Not Yet Recruiting
5
University of California, Davis
Sacramento, California, United States, 95817
Not Yet Recruiting
6
University of Florida Clinical and Translational Science Institue
Gainesville, Florida, United States, 32610
Not Yet Recruiting
7
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
8
Nemours Children's Hospital
Orlando, Florida, United States, 32827
Actively Recruiting
9
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States, 33701
Not Yet Recruiting
10
Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
11
Indiana University Health - Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Not Yet Recruiting
12
University of Kansas Medical Center
Kansas City, Kansas, United States, 66205
Actively Recruiting
13
University of Massachusetts Memorial Medical Center
North Worcester, Massachusetts, United States, 01655
Actively Recruiting
14
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
15
Atrium Health Neurosciences Institute
Charlotte, North Carolina, United States, 28207
Actively Recruiting
16
Duke University Medical Center and Childrens Health Center
Durham, North Carolina, United States, 27710
Actively Recruiting
17
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
18
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
19
Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
20
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75235
Actively Recruiting
21
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
Denton, Texas, United States, 76208
Actively Recruiting
22
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States, 78229
Actively Recruiting
23
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
Charlottesville, Virginia, United States, 22903
Actively Recruiting
24
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
25
Children's Wisconsin
Milwaukee, Wisconsin, United States, 53226
Not Yet Recruiting
26
San Jorge Children's Hospital
San Juan, Puerto Rico, Puerto Rico, 00912
Actively Recruiting
27
FDI Clinical Research
San Juan, Puerto Rico, Puerto Rico, 00927
Actively Recruiting
Research Team
S
Syune Nersisyan, PhD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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