CTIS2023-506231-15-00招募中1 期
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 - EDG-5506-203
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 200
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 65+（—）
- 性别
- Male
入选标准
- •1. Males with a diagnosis of BMD and participation in EDG-5506-002, EDG-5506-201, or EDG-5506-202 (Note: Adolescents will be enrolled in countries with regulatory approval to enroll individuals in that age range). Participants are eligible if they complete the respective prior study visits as follows: - EDG-5506-002: Complete the final study Visit 27 [Month 24]; or, completion of the ET visit prior to Visit 27 [Month 24] - EDG-5506-201: Complete the final study visit (Cohorts 1, 2, 4, and 5: Visit 12 [Month 12]; Cohort 6: Visit 11 [Month 18]) - EDG-5506-202: Complete at least 36 weeks of open-label treatment (Visit 14 [Week 52]), 2.Willing to comply with contraception requirements described in the protocol (Section 5.3.1.), 3. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
排除标准
- •1. Medical Conditions: 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., 2. Prior/Concomitant Therapy: Receiving moderate or strong cytochrome P450 CYP3A4 inhibitors or inducers., 3. Prior/Concomitant Therapy: Receipt of an investigational drug other than sevasemten within 30 days or 5 half-lives (whichever is longer) of dosing in the present study., 4. Prior/Concomitant Therapy: Receipt of oral corticosteroids for the treatment of BMD in the previous 6 months. - Receipt of low dose (=5 mg equivalent per day) oral corticosteroids for indications other than BMD or receipt for a short duration (=10 days in previous 6 months), along with receipt of chronic inhaled/intranasal steroids, is permitted., 5. Other Exclusions: Participants who are unlikely to comply with the study protocol or, in the opinion of the Investigator, would not be a suitable candidate for participation in the study., 6. Other Exclusions: Participants with a known allergy to sevasemten or its excipients., 7. Other Exclusions: Participants who are submitted to an institution by virtue of an order of a court or government authority to comply with Section 40a No. 2 of the German Medical Products Act.
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