APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 225
- 试验地点
- 1
- 主要终点
- Modified Neuropathy Impairment Score +7 (mNIS+7)
研究概览
简要总结
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis. An open-label, single-arm, long-term follow-up extension study NCT02510261 (ALN-TTR02-006) was initiated to provide participants who completed this study with continued patisiran-LNP (lipid nanoparticle) treatment.
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
- Double (Participant, Investigator)
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
- •Male or female of 18 to 85 years of age (inclusive);
- •Have a diagnosis of FAP
- •Neuropathy Impairment Score requirement of 5-130
- •Meet Karnofsky performance status requirements
- •Have adequate complete blood counts and liver function tests
- •Have adequate cardiac function
- •Have negative serology for hepatitis B virus (HBV) and hepatitis C virus (HCV)
排除标准
- •Had a prior liver transplant or is planned to undergo liver transplant during the study period;
- •Has untreated hypo- or hyperthyroidism;
- •Has known human immunodeficiency virus (HIV) infection;
- •Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated;
- •Recently received an investigational agent or device
- •Is currently taking diflunisal, tafamidis, doxycycline, or tauroursodeoxycholic acid
研究组 & 干预措施
patisiran (ALN-TTR02)
干预措施: patisiran (ALN-TTR02) (Drug)
Sterile Normal Saline (0.9% NaCl)
干预措施: Sterile Normal Saline (0.9% NaCl) (Drug)
结局指标
主要结局
Modified Neuropathy Impairment Score +7 (mNIS+7)
时间窗: 18mo
The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
次要结局
- Neurological Impairment Score-Weakness (NIS-W) Score(18mo)
- Timed 10-meter Walk Test (10-MWT, Gait Speed)(18mo)
- Rasch-built Overall Disability Scale (R-ODS) Score(18mo)
- Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Questionnaire(18mo)
- Autonomic Symptoms Questionnaire (Composite Autonomic Symptom Score [COMPASS 31])(18mo)
- Modified Body Mass Index (mBMI)(18mo)