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approved for marketing

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

key information

study id #: NCT02592941

condition: Duchenne Muscular Dystrophy

status: approved for marketing


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.

intervention: Deflazacort

mechanism of action: Glucocorticoid to delay decline in muscle strength

results: https://clinicaltrials.gov/ct2/show/results/NCT02592941

last updated: November 22, 2018

study details

study 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.

inclusion criteria:

• Eligible Sexes:

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

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

study contacts

sponsor: PTC Therapeutics

trial center locations: United States