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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)]
Who can join
Ages 2 and older · Men
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.
About the study
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).
What is being tested
- Vamorolone (drug)
Sponsor: Catalyst Pharmaceuticals, Inc. · Participants: 250 · Started: Sep 25, 2024
Contact the study team
- Mark Wingertzahn, PhD · Phone: (305) 420-3200
Official record on ClinicalTrials.gov — NCT06564974
Locations in the U.S.
| Arizona | Phoenix Children's Hospital, Phoenix |
| California | Loma Linda University Pediatric Subspecialty Clinics, Loma Linda Children's Hospital Los Angeles, Los Angeles (Not yet recruiting) Valley Children's Hospital, Madera (Not yet recruiting) Stanford University, Palo Alto (Not yet recruiting) University of California, Davis, Sacramento |
| District of Columbia | Children's National Health System, Washington D.C. (Not yet recruiting) |
| Florida | University of Florida Clinical and Translational Science Institue, Gainesville University of Miami, Miami (Not yet recruiting) Johns Hopkins All Children's Hospital, St. Petersburg |
| Minnesota | Masonic Children's Hospital - Discovery Clinic, Minneapolis (Not yet recruiting) |
| New York | The Children's Hospital at Montefiore, The Bronx (Not yet recruiting) |
| Ohio | Cincinnati Children's Hospital Medical Center, Cincinnati |
| Texas | The University of Texas Health Science Center at San Antonio, San Antonio |
| Wisconsin | Children's Wisconsin, Milwaukee (Not yet recruiting) |
Conditions
From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.