EUCTR2018-003519-24-DE进行中（未招募）1 期
An Open-label Study to Evaluate Safety, Efficacy and Pharmacokinetics (PK) of Patisiran-LNP in Patients with Hereditary Transthyretin-mediated Amyloidosis (hATTR amyloidosis) with Disease Progression Post-Orthotopic Liver Transplant
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •-18 years of age or older
- •-Received an orthotopic liver transplant for treatment of hATTR amyloidosis at least 1 year prior
- •-Increase in polyneuropathy disability post-liver transplant as measured by polyneuropathy disability (PND) score.
- •-Karnofsky performance status of =70%
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 10
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 10
排除标准
- •-Currently taking tafamidis, doxycycline, diflunisal, or tauroursodeoxycholic acid
- •-Liver allograft rejection episodes or abnormal LFTs suggestive of possible allograft rejection in the past 6 months
- •-Abnormal liver function tests
- •-Known leptomeningeal amyloidosis
- •-New York Heart Association (NYHA) classification of >2
- •-PND score IV (wheelchair bound or bedridden)
- •- Prior organ transplant other than liver transplant
- •-Hospitalization for congestive heart failure or arrhythmia in the past 3 months
研究者
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