Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
- Conditions
- Duchenne Muscular Dystrophy
- Registration Number
- NCT02592941
- Lead Sponsor
- 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.
- 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..
Recruitment & Eligibility
- Status
- APPROVED_FOR_MARKETING
- Sex
- All
- Target Recruitment
- Not specified
- Confirmed diagnosis of Duchenne muscular dystrophy
- The patient is ≥ 5 years old
- Current on all childhood vaccinations including the chicken pox vaccine
Main
- History or current medication condition that could affect safety or poses an additional risk
- Hypersensitivity or allergic reaction to steroids or their formulations
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (42)
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, 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
Scroll for more (32 remaining)Phoenix Children's Hospital🇺🇸Phoenix, Arizona, United States