DMD-001 SUMMIT Registry Study: Long-Term Safety and Quality of Life With AGAMREE in Duchenne Muscular Dystrophy
About This Study
This 5-year observational registry study will evaluate the long-term safety and clinical impact of AGAMREE® (vamorolone), developed by Santhera Pharmaceuticals, in approximately 250 male patients aged 2 years and older with Duchenne muscular dystrophy (DMD). Conducted at around 40 sites across the United States, the study will collect annual data on growth, bone and muscle health, eye and heart health, puberty, and adverse events. Researchers will also assess patients’ quality of life using standardized questionnaires and document other routine DMD treatments.
Study Overview
- Age
- 2 years and older
- Phase
- Observational
- Sponsor
- Catalyst Pharmaceuticals
- Therapeutic Approach
- Reducing Inflammation
- Variant Requirement
- Patient has a current, active prescription for, or is on, AGAMREE.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2024-09-25
- Primary Completion (Estimated)
- 2032-02
- Study Completion (Estimated)
- 2032-02
- Enrollment (Estimated)
- 250
- Countries
- United StatesPuerto Rico
Study Requirements and Criteria
Steroid Use
Not yet provided
Inclusion Criteria
- 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).
Exclusion Criteria
Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.
Contacts and Locations
Contacts and Locations
Contact
- Name: Syune Nersisyan, PhD
- Phone Number: (305) 420-3200
- Email: [email protected]
Locations
This study has 33 locations
United States
Arizona Locations
Phoenix, Arizona, United States, 85016
Actively Recruiting
Phoenix Children's Hospital
Contact: Saunder Bernes, MD
Contact Mail: [email protected]
Contact Phone: 602 933 0956
Arkansas Locations
Little Rock, Arkansas, United States, 72202
Actively Recruiting
Arkansas Childrens Hospital
Contact: Aravindhan Veerapandiyan, MD
Contact Mail: [email protected]
Contact Phone: (501) 364-1850
California Locations
Loma Linda, California, United States, 92354
Not Yet Recruiting
Loma Linda University Pediatric Subspecialty Clinics
Contact: Monica Pizana
Contact Mail: [email protected]
Contact Phone: 909-558-5830
Los Angeles, California, United States, 90027
Not Yet Recruiting
Children's Hospital Los Angeles
Contact: Dana O'Rourke, MD
Contact Mail: [email protected]
Contact Phone: 323 660 2450
Madera, California, United States, 93636
Not Yet Recruiting
Valley Children's Hospital
Contact: Claudia Nayares
Contact Mail: [email protected]
Contact Phone: (559) 353-3000
Palo Alto, California, United States, 94305
Not Yet Recruiting
Stanford University
Contact: Jenna Klotz, MD
Contact Phone: 408 426 5590
Sacramento, California, United States, 95817
Not Yet Recruiting
University of California, Davis
Contact: Craig McDonald, MD
Contact Mail: [email protected]
Contact Phone: 916 734 7041
District of Columbia Locations
Washington D.C., District of Columbia, United States, 20010
Not Yet Recruiting
Children's National Health System
Contact: Christopher Spurney, MD
Contact Mail: [email protected]
Contact Phone: 202-476-2185
Florida Locations
Gainesville, Florida, United States, 32610
Not Yet Recruiting
University of Florida Clinical and Translational Science Institue
Contact: Julie Berthy, NP
Contact Mail: [email protected]
Contact Phone: 352 273 8700
Miami, Florida, United States, 33136
Not Yet Recruiting
University of Miami
Contact: Branden Couto
Contact Mail: [email protected]
Miami, Florida, United States, 33155
Actively Recruiting
Nicklaus Children's Hospital
Contact: Migvis Monduy, MD
Contact Mail: [email protected]
Contact Phone: 305 663 8330
Orlando, Florida, United States, 32827
Actively Recruiting
Nemours Children's Hospital
Contact: Omer Abdul Hamid, MD
Contact Mail: [email protected]
Contact Phone: 407 650 7715
St. Petersburg, Florida, United States, 33701
Not Yet Recruiting
Johns Hopkins All Children's Hospital
Contact: Susan Dvojack
Contact Mail: [email protected]
Contact Phone: 727-898-7451
Illinois Locations
Chicago, Illinois, United States, 60611
Actively Recruiting
Lurie Children's Hospital of Chicago
Contact: Nancy Kuntz, MD
Contact Mail: [email protected]
Contact Phone: 312 227 4000
Indiana Locations
Indianapolis, Indiana, United States, 46202
Actively Recruiting
Indiana University Health - Riley Hospital for Children
Contact: Marcia Felker, MD
Contact Mail: [email protected]
Contact Phone: 317 278 7364
Kansas Locations
Kansas City, Kansas, United States, 66205
Actively Recruiting
University of Kansas Medical Center
Contact: Jeffrey Statland, MD
Contact Mail: [email protected]
Contact Phone: 913 588 6970
Massachusetts Locations
North Worcester, Massachusetts, United States, 01655
Actively Recruiting
University of Massachusetts Memorial Medical Center
Contact: Stephen M. Chrzanowski, MD, PhD
Contact Mail: [email protected]
Contact Phone: (774) 441-7615
Michigan Locations
Grand Rapids, Michigan, United States, 49503
Actively Recruiting
Helen DeVos Children's Hospital
Contact: Jena Krueger, MD
Contact Mail: [email protected]
Contact Phone: 616-447-5836
Minnesota Locations
Minneapolis, Minnesota, United States, 55455
Not Yet Recruiting
Masonic Children's Hospital - Discovery Clinic
Contact: Jenny Myhre
Contact Mail: [email protected]
Contact Phone: 612-365-6777
New York Locations
The Bronx, New York, United States, 10467-2403
Not Yet Recruiting
The Children's Hospital at Montefiore
Contact: Channelle Mooyoung
Contact Mail: [email protected]
Contact Phone: (718) 920-4378
North Carolina Locations
Charlotte, North Carolina, United States, 28207
Actively Recruiting
Atrium Health Neurosciences Institute
Contact: Urvi Desai, MD
Contact Mail: [email protected]
Contact Phone: 704 468 0101
Durham, North Carolina, United States, 27710
Actively Recruiting
Duke University Medical Center and Childrens Health Center
Contact: Mai ElMallah, MD
Contact Mail: [email protected]
Contact Phone: (919) 681-3364
Ohio Locations
Cincinnati, Ohio, United States, 45229
Actively Recruiting
Cincinnati Children's Hospital Medical Center
Contact: Meghan McAnally, MD
Contact Mail: [email protected]
Contact Phone: 513 636 9285
Pennsylvania Locations
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
Contact: Dustin Paul, MD
Contact Mail: [email protected]
Contact Phone: 717-531-3828
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Children's Hospital of Philadelphia (CHOP)
Contact: Sabrina Yum, MD
Contact Mail: [email protected]
Contact Phone: 215 590 4719
Texas Locations
Dallas, Texas, United States, 75235
Actively Recruiting
University of Texas Southwestern Medical Center
Contact: Kaitlin Batley, MD
Contact Mail: [email protected]
Contact Phone: 214 456 9561
Denton, Texas, United States, 76208
Actively Recruiting
Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
Contact: Diana Castro, MD
Contact Mail: [email protected]
Contact Phone: 972 982 7411
San Antonio, Texas, United States, 78229
Actively Recruiting
The University of Texas Health Science Center at San Antonio
Contact: Matthew Wicklund, MD
Contact Mail: [email protected]
Contact Phone: 210 450 0500
Virginia Locations
Charlottesville, Virginia, United States, 22903
Actively Recruiting
University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
Contact: Rebecca Scharf, MD
Contact Mail: [email protected]
Contact Phone: 434 924 1647
Washington Locations
Seattle, Washington, United States, 98105
Actively Recruiting
Seattle Children's Hospital
Contact: Seth Perlman, MD
Contact Mail: [email protected]
Contact Phone: 206-987-0804
Wisconsin Locations
Milwaukee, Wisconsin, United States, 53226
Not Yet Recruiting
Children's Wisconsin
Contact: Rebecca Rehborg
Contact Mail: [email protected]
Contact Phone: 877-607-5280
Puerto Rico
Puerto Rico Locations
San Juan, Puerto Rico, Puerto Rico, 00912
Actively Recruiting
San Jorge Children's Hospital
Contact: Yaritza Berrios
Contact Mail: [email protected]
Contact Phone: 787-758-2525
San Juan, Puerto Rico, Puerto Rico, 00927
Actively Recruiting
FDI Clinical Research
Contact: Alexandra Montalvo Acevedo, MD
Contact Mail: [email protected]
Contact Phone: 787 722 1248
Clinical Trial Registry
NCT ID
NCT06564974This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.