Evaluation of the Effect of Gene Therapy for Wilson Disease With 64CuCl₂ PET Imaging
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Ratio of liver SUV at 20 hours to liver SUV at 1 hours after injection of Cu64
研究概览
简要总结
The primary objective of this study is to investigate the effect of gene therapy (UX704) on copper distribution and excretion in Wilson disease patients. The effect is investigated using 64Cu positron emission tomography scans combined with a CT scan.
详细描述
Patients entering this study are included from the ongoing clinical study "A Phase 1/ 2/ 3 Study of UX701 Gene Therapy in Adults With Wilson Disease" (NCT04884815). The patients will have received gene therapy as part of the clinical study. This study is an extension study that aims to specifically monitor the effect of gene therapy on copper distribution and excretion using 64Cu PET/CT.
Patients will receive 40-80 MBq 64CuCl2 intravenously and will undergo three PET/CT scans during the next 20 hours.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
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 with Wilson disease who have received gene therapy (UX701) as part of NCT04884815
- •Age above 18 years
- •Women of childbearing potential must accept to use safe contraception at the time of the PET scans: Spiral or hormonal contraception (oral pills, implants, transdermal patches, vaginal rings, or depot injections)
- •At the day of the first PET scan, a negative urine pregnancy test is required for women of childbearing potential
- •Before the first PET scan, a signed informed consent must be completed
排除标准
- •Known hypersensitivity to Cu-64, copper in general, or other components of the radiotracer formulation
- •Claustrophobia
- •Pregnancy, breastfeeding, or plans to become pregnant
结局指标
主要结局
Ratio of liver SUV at 20 hours to liver SUV at 1 hours after injection of Cu64
时间窗: From first to last scan
The liver SUV is measured as the mean of five spherical VOIs (20 mm in diameter) placed in the right liver lobe, with distance to the organ borders and large blood vessels. The ratio is a measure of whether the tracer is retained in the liver (ratio \> 1) or is excreted from the liver (ratio \< 1).
次要结局
- Gallbladder SUV at 1 hour, 6 hours and 20 hours after injection of 64Cu(From first to last scan)
- Percentage of injected dose in the blood at 1 hour, 6 hours and 20 hours after injection of 64Cu(From first to last scan)
研究者
Thomas Damgaard Sandahl
Consultant, MD, PhD
University of Aarhus