Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
NCT ID: NCT02592941
Last Updated: 2018-02-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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APPROVED_FOR_MARKETING
EXPANDED_ACCESS
Brief Summary
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Detailed Description
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Conditions
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Interventions
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Deflazacort
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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* The patient is ≥ 5 years old
* Current on all childhood vaccinations including the chicken pox vaccine
Exclusion Criteria
* Hypersensitivity or allergic reaction to steroids or their formulations
5 Years
ALL
No
Sponsors
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Parexel
INDUSTRY
Dohmen Life Science Services
OTHER
PTC Therapeutics
INDUSTRY
Responsible Party
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Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
UCLA Health
Los Angeles, California, United States
Childrens Hospital of Orange County
Orange, California, United States
UC Davis Medical Center
Sacramento, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Shriner's Hospital for Children - Tampa
Tampa, Florida, United States
Children's Healthcare of Atlanta- Scottish Rite
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Rush University Medical Center
Chicago, Illinois, United States
Shriner's Hospital for Children - Chicago
Chicago, Illinois, United States
Carle Foundation Hospital
Urbana, Illinois, United States
Riley Hospital for Children at IU Health
Indianapolis, Indiana, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Michigan Health System
Ann Arbor, Michigan, United States
Wayne State University- Children's Hospital of Michigan
Detroit, Michigan, United States
Helen DeVos Children's Hospital Neurology Clinic
Grand Rapids, Michigan, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
Las Vegas Clinic
Las Vegas, Nevada, United States
Neurosciences Institute, Neurology-Charlotte
Charlotte, North Carolina, United States
Wake Forest School of Medicine
Winston-Salem, North Carolina, United States
Oregon Health & Science University
Portland, Oregon, United States
Shriner's Hospital for Children
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Greenville Health System
Greenville, South Carolina, United States
Wesley Neurology Clinic
Cordova, Tennessee, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Children's Medical Center
Dallas, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
University of Texas Health Science Center - San Antonio
San Antonio, Texas, United States
University of Virginia Children's Hospital
Charlottesville, Virginia, United States
Children's Specialty Group
Norfolk, Virginia, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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Related Links
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www.accessdmd.com
Other Identifiers
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MP-104-CL-037
Identifier Type: -
Identifier Source: org_study_id
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