An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 1,400
- 试验地点
- 245
- 主要终点
- Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
详细描述
This is a multicenter, open-label extension, Phase 3 study in up to approximately 1400 participants from the 682884-CS2 study. Eligible participants will receive eplontersen once every 4 weeks for up to 36 months or until after eplontersen is approved and available in the site's country, whichever occurs first. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Satisfactory completion of Treatment Period and the End of Treatment Visit of the Index Study (ION-682884-CS2) OR diagnosis of ATTR-CM and satisfactory participation on ISIS 420915- CS101 study as judged by the Investigator and Sponsor.
- •Investigator is willing to treat the participant with open-label eplontersen.
- •Willingness to adhere to vitamin A supplementation per protocol.
排除标准
- •Permanently discontinued study drug administration while participating in the Index Study (ION 682884-CS2) or IST (ISIS 420915-CS101 Study).
- •Have any new condition or worsening of an existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrolment, or which could interfere with the participant participating in or completing the study, including the need for treatment with medications disallowed in the Index Study.
研究组 & 干预措施
Eplontersen
Eplontersen will be administered once every month by sub-cutaneous (SC) injection for up to 36 months or 6 months after eplontersen is approved and available in the site's country, whichever occurs first.
干预措施: Eplontersen (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
时间窗: Baseline up to 36 months
Change From Baseline in Platelet Count
时间窗: Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
时间窗: Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
时间窗: Baseline up to 36 months
Number of Participants With Adverse Events (AE) and Serious Adverse Events (SAE)
时间窗: Baseline up to 36 months
Change From Baseline in Platelet Count
时间窗: Baseline up to 36 months
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
时间窗: Baseline up to 36 months
Change From Baseline in Urine Protein Creatinine Ratio (UPCR)
时间窗: Baseline up to 36 months
次要结局
- Change From Baseline in 6-minute Walk Test (6MWT)(Baseline up to 36 months)
- Change From Baseline in Transthyretin (TTR) Serum Levels(Baseline up to 36 months)
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)(Baseline up to 36 months)
- Change From Baseline in Transthyretin (TTR) Serum Levels(Baseline up to 36 months)
- Change From Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ)(Baseline up to 36 months)