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Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02592941
Expanded Access Status : Approved for marketing
First Posted : October 30, 2015
Last Update Posted : February 28, 2018
Dohmen Life Science Services
Information provided by (Responsible Party):
PTC Therapeutics

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.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Drug: Deflazacort

Detailed Description:
Deflazacort will be supplied free of charge and shipped directly to patients or caregivers, as appropriate. Treating physicians will continue to follow their standard of care activities and procedures for management of DMD. Treating physicians participating in the expanded access program are required to collect/document any patient or caregiver reported safety events and report to the sponsor..

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Study Type : Expanded Access
Official Title: 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

Intervention Details:
  • Drug: 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 Name: DFZ

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Main Inclusion Criteria:

  • Confirmed diagnosis of Duchenne muscular dystrophy
  • The patient is ≥ 5 years old
  • Current on all childhood vaccinations including the chicken pox vaccine

Main Exclusion Criteria:

  • History or current medication condition that could affect safety or poses an additional risk
  • Hypersensitivity or allergic reaction to steroids or their formulations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02592941

  Hide Study Locations
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United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
United States, California
UCLA Health
Los Angeles, California, United States, 90095
Childrens Hospital of Orange County
Orange, California, United States, 92868
UC Davis Medical Center
Sacramento, California, United States, 95817
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Florida
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32610
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Shriner's Hospital for Children - Tampa
Tampa, Florida, United States, 33612
United States, Georgia
Children's Healthcare of Atlanta- Scottish Rite
Atlanta, Georgia, United States, 30324
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Rush University Medical Center
Chicago, Illinois, United States, 60612
Shriner's Hospital for Children - Chicago
Chicago, Illinois, United States, 60707
Carle Foundation Hospital
Urbana, Illinois, United States, 61801
United States, Indiana
Riley Hospital for Children at IU Health
Indianapolis, Indiana, United States, 46202
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Michigan
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Wayne State University- Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Helen DeVos Children's Hospital Neurology Clinic
Grand Rapids, Michigan, United States, 49503
United States, Minnesota
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
United States, Nevada
Las Vegas Clinic
Las Vegas, Nevada, United States, 89145
United States, North Carolina
Neurosciences Institute, Neurology-Charlotte
Charlotte, North Carolina, United States, 28207
Wake Forest School of Medicine
Winston-Salem, North Carolina, United States, 27157
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Shriner's Hospital for Children
Portland, Oregon, United States, 97239
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, South Carolina
Greenville Health System
Greenville, South Carolina, United States, 29615
United States, Tennessee
Wesley Neurology Clinic
Cordova, Tennessee, United States, 38018
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
Children's Medical Center
Dallas, Texas, United States, 75235
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Texas Children's Hospital
Houston, Texas, United States, 77030
University of Texas Health Science Center - San Antonio
San Antonio, Texas, United States, 78229
United States, Virginia
University of Virginia Children's Hospital
Charlottesville, Virginia, United States, 22903
Children's Specialty Group
Norfolk, Virginia, United States, 23510
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Sponsors and Collaborators
PTC Therapeutics
Dohmen Life Science Services

Additional Information:
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Responsible Party: PTC Therapeutics Identifier: NCT02592941     History of Changes
Other Study ID Numbers: MP-104-CL-037
First Posted: October 30, 2015    Key Record Dates
Last Update Posted: February 28, 2018
Last Verified: February 2018

Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Anti-Inflammatory Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs