2024-511656-41-00招募中2 期
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 4
- 主要终点
- Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogram
研究概览
简要总结
To assess the long-term safety and tolerability of BMN 351 in participants with DMD
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants must have completed 351-201 or 351-203 studies without permanent discontinuation of the investigational medicinal product (IMP) or withdrawal from the study
- •Currently receiving treatment with oral corticosteroids, on a stable dose regimen during 351-201, and must remain on a consistent dose regimen throughout 351-202 or 351-203 except for modifications to accommodate changes in weight
- •Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active
排除标准
- •Have known coagulation disorder
研究组 & 干预措施
Exon 51 specific phosphorothioate oligonucleotide
Test
干预措施: Exon 51 specific phosphorothioate oligonucleotide (Drug)
结局指标
主要结局
Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogram
Incidence of adverse events/SAEs/AESI, physical examination, safety laboratory test parameters, ECG parameters, echocardiogram
次要结局
- Timed 4SC at baseline and subsequent Q24W visits
- SV95C (4-week monitoring periods preceding baseline and subsequent Q24W visits)
- PUL 2.0 at baseline and subsequent Q24W visits
- NSAA (total score and selected individual components, i.e., timed 10MWRT and TTR) at baseline and subsequent Q24W visits
研究者
Clinical Trial Information Desk
Scientific
Biomarin Pharmaceutical Inc.
研究点 (4)
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