New study tracks long-term safety of DMD drug vamorolone in boys

NCT ID NCT06564974

Recruiting now Disease control Sponsor: Catalyst Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 21, 2026 · Updated 2 times

Summary

This study follows 250 boys (ages 2 and older) with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety and real-world benefits of this treatment.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

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Locations

Arkansas Childrens Hospital
RECRUITING

Little Rock, Arkansas, 72202, United States
Atrium Health Neurosciences Institute
RECRUITING

Charlotte, North Carolina, 28207, United States
Children's Hospital Los Angeles
NOT_YET_RECRUITING

Los Angeles, California, 90027, United States
Children's Hospital of Philadelphia (CHOP)
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Children's National Health System
NOT_YET_RECRUITING

Washington D.C., District of Columbia, 20010, United States
Children's Wisconsin
NOT_YET_RECRUITING

Milwaukee, Wisconsin, 53226, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Duke University Medical Center and Childrens Health Center
RECRUITING

Durham, North Carolina, 27710, United States
FDI Clinical Research
RECRUITING

San Juan, Puerto Rico, 00927, Puerto Rico
Helen DeVos Children's Hospital
RECRUITING

Grand Rapids, Michigan, 49503, United States
Indiana University Health - Riley Hospital for Children
RECRUITING

Indianapolis, Indiana, 46202, United States
Johns Hopkins All Children's Hospital
NOT_YET_RECRUITING

St. Petersburg, Florida, 33701, United States
Loma Linda University Pediatric Subspecialty Clinics
NOT_YET_RECRUITING

Loma Linda, California, 92354, United States
Lurie Children's Hospital of Chicago
RECRUITING

Chicago, Illinois, 60611, United States
Masonic Children's Hospital - Discovery Clinic
NOT_YET_RECRUITING

Minneapolis, Minnesota, 55455, United States
Nemours Children's Hospital
RECRUITING

Orlando, Florida, 32827, United States
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
RECRUITING

Denton, Texas, 76208, United States

Contact Email: •••••@•••••
Nicklaus Children's Hospital
RECRUITING

Miami, Florida, 33155, United States
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
RECRUITING

Hershey, Pennsylvania, 17033, United States
Phoenix Children's Hospital
RECRUITING

Phoenix, Arizona, 85016, United States
San Jorge Children's Hospital
RECRUITING

San Juan, Puerto Rico, 00912, Puerto Rico
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105, United States
Stanford University
NOT_YET_RECRUITING

Palo Alto, California, 94305, United States
The Children's Hospital at Montefiore
NOT_YET_RECRUITING

The Bronx, New York, 10467-2403, United States
The University of Texas Health Science Center at San Antonio
RECRUITING

San Antonio, Texas, 78229, United States
University of California, Davis
NOT_YET_RECRUITING

Sacramento, California, 95817, United States
University of Florida Clinical and Translational Science Institue
NOT_YET_RECRUITING

Gainesville, Florida, 32610, United States
University of Kansas Medical Center
RECRUITING

Kansas City, Kansas, 66205, United States
University of Massachusetts Memorial Medical Center
RECRUITING

North Worcester, Massachusetts, 01655, United States
University of Miami
NOT_YET_RECRUITING

Miami, Florida, 33136, United States
University of Texas Southwestern Medical Center
RECRUITING

Dallas, Texas, 75235, United States
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
RECRUITING

Charlottesville, Virginia, 22903, United States
Valley Children's Hospital
NOT_YET_RECRUITING

Madera, California, 93636, United States

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