An Open-Label Extension Study to Assess the Long-term Effect of EDG-5506 on Safety, Biomarkers, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 入组人数
- 260
- 试验地点
- 81
- 主要终点
- Number of adverse events in those treated with sevasemten
研究概览
简要总结
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
详细描述
This is an open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of sevasemten. EDG-5506-203 MESA will provide continued access to sevasemten treatment to participants with Becker muscular dystrophy who were previously enrolled in EDG-5506-002 ARCH, completed EDG-5506-201 CANYON and GRAND CANYON, or completed EDG-5506-202 DUNE.
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
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •1. Males with a diagnosis of BMD and participation in EDG-5506-002 ARCH, EDG-5506-201 CANYON and GRAND CANYON, or EDG-5506-202 DUNE. Participants are eligible if they complete the respective prior study visits as follows:
- •EDG-5506-002 ARCH: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24]
- •EDG-5506-201 CANYON and GRAND CANYON: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18])
- •EDG-5506-202 DUNE: Complete at least 36 weeks of open-label treatment (Visit 14 [Week 52])
排除标准
- •Any clinically significant changes during or following participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 that would affect the potential safety of the participant to receive sevasemten.
- •Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers.
- •Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study.
- •Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months.
研究组 & 干预措施
Treatment
Drug: Sevasemten
干预措施: Sevasemten (Drug)
结局指标
主要结局
Number of adverse events in those treated with sevasemten
时间窗: 55 Months
Severity of adverse events in those treated with sevasemten
时间窗: 55 Months
Number of adverse events in those treated with sevasemten
时间窗: 37 Months
Severity of adverse events in those treated with sevasemten
时间窗: 37 Months
次要结局
- Incidence of treatment-emergent abnormal clinical chemistry laboratory test results(54 Months)
- Incidence of treatment-emergent abnormal hematology laboratory test results(54 Months)
- Incidence of treatment-emergent abnormal clinical chemistry laboratory test results(36 Months)
- Incidence of treatment-emergent abnormal hematology laboratory test results(36 Months)