Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy
1 other identifier
expanded_access
N/A
1 country
42
Brief Summary
The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
42 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 29, 2015
CompletedFirst Posted
Study publicly available on registry
October 30, 2015
CompletedFebruary 28, 2018
February 1, 2018
October 29, 2015
February 26, 2018
Conditions
Interventions
Deflazacort, a glucocorticoid with anti-inflammatory and immunosuppressive effects, is used in treating a variety of diseases. Pharmacologically it is an inactive pro-drug which is metabolized immediately to the active metabolite, 21 desacetyl-DFZ. The elimination of this metabolite is primarily via the urine in humans. Its potency is approximately 70 to 90% of prednisone and 6 mg of deflazacort has approximately the same anti-inflammatory potency as 5 mg of prednisolone or prednisone.
Eligibility Criteria
You may qualify if:
- Confirmed diagnosis of Duchenne muscular dystrophy
- The patient is ≥ 5 years old
- Current on all childhood vaccinations including the chicken pox vaccine
You may not qualify if:
- History or current medication condition that could affect safety or poses an additional risk
- Hypersensitivity or allergic reaction to steroids or their formulations
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- PTC Therapeuticslead
- Parexelcollaborator
- Dohmen Life Science Servicescollaborator
Study Sites (42)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCLA Health
Los Angeles, California, 90095, United States
Childrens Hospital of Orange County
Orange, California, 92868, United States
UC Davis Medical Center
Sacramento, California, 95817, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
Nicklaus Children's Hospital
Miami, Florida, 33155, United States
Shriner's Hospital for Children - Tampa
Tampa, Florida, 33612, United States
Children's Healthcare of Atlanta- Scottish Rite
Atlanta, Georgia, 30324, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Shriner's Hospital for Children - Chicago
Chicago, Illinois, 60707, United States
Carle Foundation Hospital
Urbana, Illinois, 61801, United States
Riley Hospital for Children at IU Health
Indianapolis, Indiana, 46202, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
University of Michigan Health System
Ann Arbor, Michigan, 48109, United States
Wayne State University- Children's Hospital of Michigan
Detroit, Michigan, 48201, United States
Helen DeVos Children's Hospital Neurology Clinic
Grand Rapids, Michigan, 49503, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101, United States
Las Vegas Clinic
Las Vegas, Nevada, 89145, United States
Neurosciences Institute, Neurology-Charlotte
Charlotte, North Carolina, 28207, United States
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Shriner's Hospital for Children
Portland, Oregon, 97239, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Greenville Health System
Greenville, South Carolina, 29615, United States
Wesley Neurology Clinic
Cordova, Tennessee, 38018, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Children's Medical Center
Dallas, Texas, 75235, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
University of Texas Health Science Center - San Antonio
San Antonio, Texas, 78229, United States
University of Virginia Children's Hospital
Charlottesville, Virginia, 22903, United States
Children's Specialty Group
Norfolk, Virginia, 23510, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 29, 2015
First Posted
October 30, 2015
Last Updated
February 28, 2018
Record last verified: 2018-02