DMD-001 SUMMIT Registry Study: Long-Term Safety and Quality of Life With AGAMREE in Duchenne Muscular Dystrophy
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.