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

Recruiting now

Conditions studied: Duchenne Muscular Dystrophy

In brief

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).

Key facts

Study ID

NCT06564974

Run by

Catalyst Pharmaceuticals, Inc.

People needed

250

Starts

2024-09-25

Expected to finish

2032-02-01

Last updated by the study team

2026-07-20

Who can join

Age: 2 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
• Male patients at least 2 years old.
• 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).
• Patient has a current, active prescription for, or is on, AGAMREE®.

You may not qualify if…

• Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.

Where it is running

• FDI Clinical Research — San Juan, Puerto Rico, Puerto Rico (enrolling)
• Arkansas Childrens Hospital — Little Rock, Arkansas, United States (enrolling)
• Atrium Health Neurosciences Institute — Charlotte, North Carolina, United States (enrolling)
• Duke University Medical Center and Childrens Health Center — Durham, North Carolina, United States (enrolling)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
• Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute — Hershey, Pennsylvania, United States (enrolling)
• Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania, United States (enrolling)
• University of Texas Southwestern Medical Center — Dallas, Texas, United States (enrolling)
• Neurology Rare Disease Center - Neurology & Neuromuscular Care Center — Denton, Texas, United States (enrolling)
• The University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States (enrolling)
• University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center — Charlottesville, Virginia, United States (enrolling)
• Seattle Children's Hospital — Seattle, Washington, United States (enrolling)
• San Jorge Children's Hospital — San Juan, Puerto Rico, Puerto Rico (enrolling)
• Phoenix Children's Hospital — Phoenix, Arizona, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Nemours Children's Hospital — Orlando, Florida, United States (enrolling)
• Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Indiana University Health - Riley Hospital for Children — Indianapolis, Indiana, United States (enrolling)
• University of Kansas Medical Center — Kansas City, Kansas, United States (enrolling)
• University of Massachusetts Memorial Medical Center — North Worcester, Massachusetts, United States (enrolling)
• Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States (enrolling)
• Loma Linda University Pediatric Subspecialty Clinics — Loma Linda, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Valley Children's Hospital — Madera, California, United States
• Stanford University — Palo Alto, California, United States

Full record on ClinicalTrials.gov

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