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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
October 29, 2015
October 30, 2015
August 18, 2017
 
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
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.
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..
Expanded Access
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
Not Provided
 
Available
Contact: Call Center 1-844-800-4DMD (4363)
United States
 
 
NCT02592941
PTC Therapeutics
PTC Therapeutics
  • Parexel
  • Dohmen Life Science Services
Not Provided
PTC Therapeutics
August 2017