An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of ALXN2220 in Adult Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 1,180
- 试验地点
- 195
- 主要终点
- Percentage of participants with TEAEs
研究概览
简要总结
The purpose of Study ALXN2220-ATTRCM-302 is to evaluate the long-term safety, tolerability and efficacy of ALXN2220 in adult participants with ATTR-CM treated with ALXN2220 in addition to SoC who have completed Study ALXN2220-ATTRCM-301.
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
盲法说明
Study 302 is a single arm, OLE study. All participants will receive open-label ALXN2220 upon entry into the OLE study. No IMP masking or blinding procedures will be implemented or maintained during the OLE study.
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Inclusion Criteria
- •Type of Participant: Participants who have completed the Blinded Treatment Period (including the PC visit) of Study
- •Sex and Contraceptive/Barrier Requirements: Male and/or female (according to their reproductive organs and functions assigned by chromosomal complement).
- •Informed Consent: Participant, or legally authorized representative where relevant and allowed by law, is willing and able to comply with all study requirements and to provide signed informed consent.
排除标准
- •Participants who permanently discontinued study intervention in Study 301
- •Participants who withdrew full consent during Study
- •Medical Conditions: Suspected or known intolerance/allergy to proteins or any components of ALXN
- •Has had a heart transplant or permanent CMAD implantation. Has any new or worsening of existing medical or psychological condition or risk factor that in the opinion of the Investigator or Sponsor might jeopardize participant's safety or compliance with the protocol, or pose any additional risk for the participant, or confound the assessment of the participant or outcome of the study.
- •Prior/Concurrent/Future Clinical Study Experience: Current or future participation in another investigational clinical study or intake of investigational drug within 30 calendar days or 5 half-lives of the study intervention, whichever is longer, before signing the ICF, except for study intervention used in Study 301.
研究组 & 干预措施
ALXN2220
Eligible participants will receive ALXN2220 administered IV q4w for a minimum of 12 months and up to 24 months or until participant has access to commercially available ALXN2220 for ATTR-CM, whichever occurs first.
干预措施: ALXN2220 (Drug)
结局指标
主要结局
Percentage of participants with TEAEs
时间窗: Up to 24 months
To evaluate the long-term safety and tolerability of ALXN2220 in participants with ATTR-CM
次要结局
- Time to CVM(Up to 24 months)
- Total occurrence of ACM and CV clinical events(Up to 24 months)
- Total occurrence of CV clinical events(Up to 24 months)
- Time to ACM(Up to 24 months)
- Time to first CV clinical event or ACM(Up to 24 months)
- Change from baseline in KCCQ-OS by visit(Up to 24 months)
- Change from baseline in 6MWT by visit(Up to 24 months)
- ADA incidence, response categories, and titer(Up to 24 months)
- NAb incidence and response categories(Up to 24 months)