A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 67
- 试验地点
- 1
- 主要终点
- Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline
研究概览
简要总结
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
- •PND score of I-IIIB at baseline.
- •Exposure to commercial patisiran in one of the 3 cohorts:
- •Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
- •Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
- •Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.
排除标准
- •New York Heart Association (NYHA) heart failure classification ≥3
- •Karnofsky Performance Status (KPS) <60%
- •Unstable congestive heart failure (CHF)
- •Known primary amyloidosis (AL) or leptomeningeal amyloidosis
- •Prior major organ transplant
- •Previously received patisiran
- •Previous treatment with a TTR silencing therapy
研究组 & 干预措施
Patisiran Prospective Cohort
Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.
干预措施: Patisiran (Drug)
Patisiran Mixed Cohort
Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.
干预措施: Patisiran (Drug)
Patisiran Retrospective Cohort
Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.
干预措施: Patisiran (Drug)
结局指标
主要结局
Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline
时间窗: Baseline, Month 12
PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.
次要结局
未报告次要终点