NCT06672237招募中3 期
MAGNITUDE-2: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 28
- 主要终点
- Modified Neuropathy Impairment Score +7 (mNIS+7)
研究概览
简要总结
This study will be conducted to evaluate the efficacy and safety of a single dose of nexiguran ziclumeran (NTLA-2001) compared to placebo in participants with ATTRv-PN.
详细描述
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 60 participants, who will be randomized 1:1 to receive a single infusion of either nexiguran ziclumeran or placebo. To ensure all participants will have the potential to receive nexiguran ziclumeran, participants will have the option to cross over to the opposite study arm at Month 12 or Month 18, depending on study criteria.
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of ATTRv-PN
- •Karnofsky Performance Status (KPS) ≥ 60
排除标准
- •Other causes of amyloidosis (amyloidosis caused by non-TTR protein)
- •Other known causes of sensorimotor or autonomic neuropathy
- •Diabetes mellitus
- •New York Heart Association Class III or IV heart failure
- •Liver failure
- •Hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- •Prior receipt of a TTR silencer (Small interfering RNA (siRNA) or Antisense oligonucleotides (ASOs))
- •Estimated Glomerular Filtration Rate < 30 mL/min/1.73 m2
- •Unable or unwilling to take vitamin A supplementation for the duration of the study
- •History of liver disease
研究组 & 干预措施
nexiguran ziclumeran
Experimental
nexiguran ziclumeran 55 mg by single IV infusion
干预措施: nexiguran ziclumeran (Biological)
Normal Saline
Placebo Comparator
Placebo; Normal saline (0.9% NaCl) by single IV infusion
干预措施: Normal Saline as Placebo (Drug)
结局指标
主要结局
Modified Neuropathy Impairment Score +7 (mNIS+7)
时间窗: 18 Months
Serum TTR
时间窗: 29 Days
次要结局
- Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire(18 Months)
- Modified Body Mass Index (mBMI)(18 Months)
- Serum TTR(18 Months)
研究者
研究点 (28)
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相关资讯
Intellia'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