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
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 入组人数
- 18
- 试验地点
- 6
研究概览
简要总结
This open-label extension study aims to evaluate the long-term safety and tolerability of weekly BMN 351 infusions, as well as to assess the effect of BMN 351 on physical function, in participants with DMD who participated in the 351-201 study.
详细描述
This Phase 2, multi-center, open-label extension study is designed to assess the long-term safety, tolerability, and functional efficacy of weekly intravenous doses of BMN 351 administered to participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping in the Phase 1/2 study, 351-201. Up to 18 participants ages 4 through 23 at baseline will enroll in the trial after completing 351-201.
The first visit for this study is the same as the final visit of 351-201. To be eligible for this study, potential participants must satisfy the eligibility criteria described in the protocol.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants must have completed 351-201 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
- •Transition to the equivalent dose of vamorolone is permitted in 351-202 where approved in participating countries.
- •Willing and able to adhere to the study visit schedule and other protocol requirements
- •Willing to use contraception (sexually mature males) throughout the study and for 90 days after the final dose, if sexually active
- •Contraceptive use by males should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure
- •Willing and able to provide written, signed informed consent as parent or guardian after the nature of the study has been explained and prior to performance of any research-related procedure
排除标准
- •Have known coagulation disorder
- •Are taking any prohibited medications
- •any approved exon skipping therapy within 12 weeks prior to baseline or with any gene therapy for the treatment of DMD at any time
- •anti-coagulants, anti-thrombotics, or anti-platelet agents
- •immunosuppressants
研究组 & 干预措施
BMN 351
Participants from 351-201 enrolling in 351-202 will initially receive BMN 351 at the dose level at which they completed 351-201. Once all 351-201 participants have completed their Week 25 visit in 351-202, available safety and PD data will be analyzed and a single optimal dose level will be selected for 351-202. All participants from 351-201 will transition to that dose level at their next applicable visit.
干预措施: BMN 351 (Drug)
结局指标
主要结局
未指定
次要结局
- To evaluate the effect of BMN 351 on physical function(Change from baseline and subsequent 24-week incremental visits)