MyocardON-TTR - Myocardial Effects in Patients With Hereditary Transthyretin-mediated Amyloidosis With Polyneuropathy Treated With Patisiran or Vutrisiran
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 20
- 试验地点
- 5
- 主要终点
- Difference in longitudinal relaxation time (T1) per mapping between M0 and M24
研究概览
简要总结
ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).
Patisiran and vutrisiran have shown protective effects on the progression of neurological damage.
The effects of Patisiran or vutrisiran on the heart remain incompletely understood. The aim of this study is to better understand the morphological and functional cardiac consequences in ATTRv patients with stage 1 or 2 polyneuropathy with a mixed form treated with Patisiran or vutrisiran
详细描述
ATTRv amyloidosis is a systemic disease with two clinical forms, neurological and cardiological, which are sometimes combined (so-called mixed forms).
Patisiran and vutrisiran have shown protective effects on the progression of neurological damage.
The effects of Patisiran or vutrisiran on the heart remain incompletely understood.
During their therapeutic management, including the prescription of Patisiran or vutrisiran, the routine examinations carried out at the inclusion, one and two year later will allow us to observe the consequences on myocardial activity during the routine consultation after 1 and 2 years of treatment.
Examinations are : clinical and biological exams, EKC, echocardiography, cardiac MRI and scintigraphy.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 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
- •Patients aged 18 years or older
- •Patients with hereditary transthyretin amyloidosis (ATTRv) with stage 1 or 2 polyneuropathy
- •Patient not previously treated for ATTRv
- •Patients for whom treatment with patisiran or vutrisiran has been initiated by a hospital neurologist in accordance with recommendations for a minimum of 24 months.
- •Patients with NYHA stage 1 and 2 cardiac disease.
- •Beneficiary of a social security scheme
- •Person who does not object to his/her participation in the research
排除标准
- •Patients treated with Tafamidis simultaneously with patisiran or vutrisiran
- •Adults under legal protection (legal guardianship, curatorship, guardianship), persons deprived of liberty.
- •Contraindications to the explorations provided for in the protocol: claustrophobia, metallic implant contraindicating MRI, woman of childbearing age
结局指标
主要结局
Difference in longitudinal relaxation time (T1) per mapping between M0 and M24
时间窗: 24 months
Longitudinal relaxation time (T1) is the process by which the net magnetization (M) grows/returns to its initial maximum value (Mo) parallel to Bo in a MRI. Myocardial T1 depends on the pulse sequence, cardiac cycle as well as other factors and increases at higher magnetic field strength. T1-mapping can detect a variety of myocardial pathologies, where it shows increased values. Because of variations between scanners the primary use of a local reference range is recommended and if a local reference range is not available quantitative results should not be clinically reported. An intermediate analysis will be done at 12 months
次要结局
- Difference in Compass31 questionnaire score between M0 and M24(24 months)
- Difference in distance obtained in the 6-minute walking test between M0 and M24(24 months)
- Difference in Perugini Grading Score between M0 and M24(24 months)
- Difference in the value of the global longitudinal strain between M0 and M24(24 months)
- Difference in Kansas City Cardiomyopathy Questionnaire score between M0 and M24(24 months)
- Difference in the value of the global strain of the right ventricular free wall between M0 and M124(24 months)
- Difference in the value of the global left atrial longitudinal strain between M0 and M24(24 months)
- Evolution of myocardial work between M0 and M24(24 months)