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

Expanded access is currently available for this treatment.
Verified August 2017 by PTC Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT02592941
First Posted: October 30, 2015
Last Update Posted: August 18, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
Parexel
Dohmen Life Science Services
Information provided by (Responsible Party):
PTC Therapeutics
  Purpose
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 Intervention
Duchenne Muscular Dystrophy Drug: Deflazacort

Study Type: Expanded Access     What is 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

Resource links provided by NLM:


Further study details as provided by PTC Therapeutics:

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
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..
  Eligibility

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.


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

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
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT02592941


Contacts
Contact: Call Center 1-844-800-4DMD (4363)

  Hide Study Locations
Locations
United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Contact: Saunder Bernes, MD         
United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Contact: Vikki Stefans, MD         
United States, California
UCLA
Los Angeles, California, United States, 90095
Contact: Perry Shieh, MD         
UC Davis Medical Center
Sacramento, California, United States, 95817
Contact: Craig McDonald, MD         
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Contact: Joanne Janas, MD         
United States, District of Columbia
Children's National Medical Center
Washington, D.C., District of Columbia, United States, 20010
Contact: Mathula Thangarajh, MD         
United States, Florida
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32610
Contact: Barry Byrne, MD         
Shriner's Hospital for Children - Tampa
Tampa, Florida, United States, 33612
Contact: Raymond Fernandez, MD         
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30324
Contact: Sumit Verma, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Contact: Nancy Kuntz, MD         
Rush University Medical Center
Chicago, Illinois, United States, 60612
Contact: Peter Heydemann, MD         
Shriner's Hospital for Children - Chicago
Chicago, Illinois, United States, 60707
Contact: Kenneth Silver, MD         
Carle Foundation Hospital
Urbana, Illinois, United States, 61801
Contact: Robert Cranston, MD         
United States, Indiana
Riley Hospital for Children at IU Health
Indianapolis, Indiana, United States, 46202
Contact: Marcia Felker, MD         
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
Contact: Katherine Mathews, MD         
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Contact: Jeffrey Statland, MD         
United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
Contact: Kathryn Wagner, MD         
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Contact: Basil Darras, MD         
United States, Michigan
University of Michigan Health System
Ann Arbor, Michigan, United States, 48109
Contact: Erin Neil, MD         
Wayne State University- Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Contact: Huiyuan Jiang, MD         
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Contact: Steven DeRoos, MD         
United States, Minnesota
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
Contact: Randal Richardson, MD         
United States, Nevada
Las Vegas Clinic
Las Vegas, Nevada, United States, 89145
Contact: Jonathan McKinnon, MD         
United States, North Carolina
Neurosciences Institute, Neurology-Charlotte
Charlotte, North Carolina, United States, 28207
Contact: Elena Bravver, MD         
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Contact: Chafic Karam, MD         
Shriner's Hospital for Children
Portland, Oregon, United States, 97239
Contact: Erika Finanger, MD         
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Contact: Matthew Wicklund, MD         
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
Contact: Hoda Abdel-Hamid, MD         
United States, South Carolina
Greenville Health System
Greenville, South Carolina, United States, 29615
Contact: Addie Hunnicutt, MD         
United States, Tennessee
Wesley Neurology Clinic
Memphis, Tennessee, United States, 38104
Contact: Tulio Bertorini, MD         
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Contact: William Burnette, MD         
United States, Texas
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Contact: Warren Marks, MD         
Texas Children's Hospital
Houston, Texas, United States, 77030
Contact: Farida Abid, MD         
University of Texas Health Science Center - San Antonio
San Antonio, Texas, United States, 78229
Contact: Ratna Bhavaraju-Sanka, MD         
United States, Virginia
University of Virginia Children's Hospital
Charlottesville, Virginia, United States, 22903
Contact: Rebecca Scharf, MD         
Children's Specialty Group
Norfolk, Virginia, United States, 23510
Contact: L. Mathew Frank, MD         
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Contact: Cristian Ionita, MD         
Sponsors and Collaborators
PTC Therapeutics
Parexel
Dohmen Life Science Services
  More Information

Additional Information:
Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT02592941     History of Changes
Other Study ID Numbers: MP-104-CL-037
First Submitted: October 29, 2015
First Posted: October 30, 2015
Last Update Posted: August 18, 2017
Last Verified: August 2017

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