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

PTC Therapeutics

Status

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: Deflazacort

Study type: Expanded Access

Funder types: Other; Industry

Identifiers

NCT02592941

MP-104-CL-037

Details and patient eligibility

About

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.

Full 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..

Sex: All

Ages: 5+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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