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

• Status: Approved for marketing
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

Details

• Lead Sponsor: Marathon Pharmaceuticals, LLC; PTC Therapeutics
• Collaborators: Dohmen Life Science Services; Parexel
• Treatments: Deflazacort

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