An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 不适用
- 状态
- Approved For Marketing
- 试验地点
- 42
研究概览
简要总结
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.
详细描述
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..
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 5 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of Duchenne muscular dystrophy
- •The patient is ≥ 5 years old
- •Current on all childhood vaccinations including the chicken pox vaccine
排除标准
- •History or current medication condition that could affect safety or poses an additional risk
- •Hypersensitivity or allergic reaction to steroids or their formulations