NCT06128629进行中（未招募）3 期
MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 1,200
- 试验地点
- 124
- 主要终点
- Composite outcome of cardiovascular (CV) mortality and CV events
研究概览
简要总结
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
详细描述
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnosis of ATTR amyloidosis with cardiomyopathy
- •Medical history of heart failure (HF)
- •Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
- •Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL
排除标准
- •New York Heart Association (NYHA) Class IV HF
- •Polyneuropathy Disability score of IV (confined to wheelchair or bed)
- •Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- •History of active malignancy within 3 years prior to screening
- •RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
- •Initiation of tafamidis or acoramidis within 56 days prior to study dosing
- •Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2
- •Liver failure
- •Uncontrolled blood pressure
- •Unable or unwilling to take vitamin A supplementation for the duration of the study
研究组 & 干预措施
Placebo
Placebo Comparator
Single IV infusion of normal saline
干预措施: Placebo (Drug)
NTLA-2001
Experimental
Single intravenous (IV) infusion of NTLA-2001
干预措施: NTLA-2001 (Biological)
结局指标
主要结局
Composite outcome of cardiovascular (CV) mortality and CV events
时间窗: Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months
次要结局
- Change in baseline to month 18 in serum TTR(Baseline, Month 18)
- Change from baseline to month 18 in KCCQ-OS score(Baseline, Month 18)
研究者
研究点 (124)
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相关资讯
Intellia's NTLA-2001 Shows Promise in Early ATTR-CM Trial• Intellia Therapeutics' NTLA-2001 demonstrates potential in stabilizing or improving outcomes for transthyretin amyloidosis with cardiomyopathy (ATTR-CM) patients.
• Phase I study data reveals a median five-meter improvement in the six-minute walk test after one year of NTLA-2001 treatment.
• 92% of patients experienced either improvement or maintenance in their New York Heart Association (NYHA) class, indicating heart failure stabilization.
• NTLA-2001, a CRISPR-based therapy, aims to reduce misfolded transthyretin (TTR) protein production, with a mean TTR reduction of 90% observed after one year.last yearIntellia's Nexiguran Ziclumeran Shows Promising Phase 1 Results in ATTR Amyloidosis• Intellia's nexiguran ziclumeran (nex-z) demonstrates rapid and durable TTR reduction in ATTR amyloidosis patients after a single dose.
• Phase 1 data indicates disease stabilization or improvement in cardiac markers for ATTR-CM patients and neuropathy for ATTRv-PN patients.
• The CRISPR-based therapy shows a favorable safety profile with mainly mild to moderate infusion-related reactions.
• Intellia is advancing nex-z into Phase 3 trials based on the hypothesis that greater TTR reduction leads to greater clinical benefit.last yearIntellia's CRISPR Therapy Nexiguran Ziclumeran Shows Promise in ATTR Amyloidosis Phase 1 Trial• Intellia's nexiguran ziclumeran (nex-z) demonstrates rapid and durable TTR reduction in ATTR amyloidosis patients, suggesting potential for significant clinical benefit.
• The Phase 1 trial reveals stabilization or improvement in cardiac markers for ATTR-CM patients and neuropathy measures for ATTRv-PN patients after nex-z treatment.
• A single dose of nex-z maintains persistently low TTR levels for over two years in some patients, with a favorable safety and tolerability profile observed.
• These positive results support the ongoing Phase 3 trials evaluating nex-z in larger ATTR-CM and ATTRv-PN populations, reinforcing the hypothesis that greater TTR reduction leads to better outcomes.last year