Effect of Radiotherapy on ATTR Cardiac Amyloidosis : a Proof of Concept Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 36
- 试验地点
- 2
- 主要终点
- Assessment of the degree of amyloid
研究概览
简要总结
Cardiac amyloidosis is responsible for significant morbidity associated with heart failure, and carries a poor prognosis. Currently there are very limited treatment options for this condition. Radiotherapy has been used successfully to treat amyloidosis elsewhere in the body, however has not been tried in cardiac amyloidosis. Therefore this study aims to assess the effect of radiotherapy on cardiac amyloidosis, to evaluate whether it can successfully reduce the burden of amyloid deposits in the myocardium as assessed by 18F-Amyloid PET.
详细描述
The intervention will involve administration of external beam radiotherapy (5 fractions of 2Gy) focused to the heart.
Measurements of effect will be assessed at 12 weeks by:
Amyloid PET Cardiac MRI with administration of gadolinium and ultrasound A blood venous sample (cardiac biomarkers) Quality of life assessments
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 65 Years 至 —（Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age >65 y.o.
- •Dyspnoea on exertion (NYHA II or more).
- •Stable elevated cardiac enzymes (ultra sensitive Troponin T > 14 ng/L on consecutive sampling or BNP > 100pg/mL)
- •A positive 99mTc-DPD cardiac scintigraphy (Grade 2 and 3) suggesting an ATTR or wt amyloidosis.
- •Additional imaging also compatible with cardiac amyloidosis (cardiac ultrasound showing basal to apical longitudinal strain gradient and magnetic resonance imaging with elevated T1 value or extracellular volume).
- •Compliance with the informed consent as attested by its signature.
- •Positive baseline 18F-Florbetapir imaging, as assessed visually and quantitatively by a Tissue to Background Ratio > 1.45
排除标准
- •Positive serum protein immunoelectrophoresis with monoclonal gammapathy.
- •Previous external beam radiotherapy including the chest.
- •Claustrophobia
- •Presence of internal non-MR compatible devices
- •Creatinine glomerular filtration rate < 30 ml/min
- •Oncologic disease (excluding skin cancer) active or in remission from less than 5 years
结局指标
主要结局
Assessment of the degree of amyloid
时间窗: 12 weeks
Assess intra-individual change in a quantitative measure of amyloid deposits on 18F-Amyloid (Standard Uptake Value ratio) SUVR between amyloid PET scans before and after low dose RT
次要结局
- Modification in echocardiographic global longitudinal strain(6 months)
- Modification in cardiac biomarker (ultrasensitive troponin T and BNP blood levels) features of cardiac amyloidosis(6 months)
- Safety and adverse event associated with cardiac low dose RT(6 months)
- Modification of quality of life(6 months)
- Modification in MRI extracellular volumes and T1 values(12 weeks)
研究者
Philippe Meyer
Principal Investigator
University Hospital, Geneva