Evaluation of 124I-p5+14 Injection as an Imaging Agent for the Detection of Systemic Amyloidosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 57
- 试验地点
- 1
- 主要终点
- Organ-specific radioactivity dosimetry (Part 1).
研究概览
简要总结
This is a single-center, exploratory, Phase 1 Positron Emission Tomography/x-ray Computed Tomography (PET/CT) imaging study to detect amyloidosis that will enroll patients with a confirmed diagnosis of systemic amyloidosis. The purpose of this exploratory trial is to assess the safety and efficacy of 124I-p5+14 Injection at a single-injection dose adequate for imaging amyloid deposits by using PET/CT imaging in subjects with confirmed systemic Immunoglobulin Light Chain-associated Amyloidosis (AL), Transthyretin-associated Amyloidosis (ATTR), Leukocyte Chemotactic Factor 2-associated Amyloidosis (ALect2) as well as other types.
详细描述
The rationale for this study is the discovery of a synthetic polypeptide, designated p5+14, a synthetic 45 amino acid peptide that binds many forms of amyloid, including human AL-, ATTR- and ALect2-associated amyloid, as well as human and murine serum amyloid protein A-associated (AA) amyloid. In preclinical studies, using SPECT and PET imaging, as well as microautoradiography, it has been shown that radioiodinated p5+14 binds rapidly and specifically to all amyloid deposits in abdominothoracic organs and tissues.
This is a single site, exploratory, open-label Phase I PET/CT imaging and dosimetry study. The investigational drug product (designated 124I-p5+14 Injection) is an amyloid-reactive synthetic peptide, p5+14 (also known as APi1832), radiolabeled with iodine-124 (I-124 or 124I). All patients enrolled in this exploratory trial will be outpatients with a confirmed diagnosis of systemic amyloidosis.
The first three patients enrolled in the trial (Part 1) will take part in a dose-escalation dosimetry study and will receive a single intravenous (IV) dose of 11.1 Megabecquerel (MBq) (0.3 millicuries (mCi); n = 1), 37 MBq (1 mCi; n = 1) or 74 MBq (2 mCi; n = 1) of 124I-p5+14 Injection for the purpose of determining estimates of organ-associated and whole body radioactive dosimetry.
The trial then will be opened to include another 54 patients who will receive a single IV bolus injection of 2 mCi 124I-p5+14 Injection. Every patient participating will receive < 2 mg of peptide p5+14. The study comprises five parts.
In Part 2, the trial will enroll SA patients with confirmed AL, ATTR, ALECT2, or other forms of amyloidosis.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
盲法说明
open-label
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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
124I-p5+14 Injection
A single-injection dose of 124I-p5+14 adequate for imaging amyloid deposits by using PET/CT imaging in subjects with confirmed systemic amyloidosis of several sub-types.
干预措施: 124I-p5+14 Injection (Drug)
结局指标
主要结局
Organ-specific radioactivity dosimetry (Part 1).
时间窗: Through 48 hours
Localization of 124I-p5+14 will be taken from PET/CT images performed at intervals during the 48 hours after injection. Organ-specific dosimetry and whole body dose measurements will be made using Olinda software (Olinda/Exp; Organ Level Internal Dose Assessment/Exponential Modeling)
次要结局
- Absolute values and changes from baseline in clinical laboratory values.(Baseline and 24 hours)
- Clinically defined amyloidosis organ involvement.(Baseline)
- Measure of background radioactivity uptake.(6 hours and 24 hours)
- Measure of radioactivity uptake by each organ(6 hours and 24 hours)
- Concentration of radiotracer in specific anatomic sites, for each subject and anatomic site(6 hours and 24 hours)
- Correlation between concentration of the radiotracer (Bq/cc) in the kidney with organ-associated clinical biomarkers.(6 hours and 24 hours)
- Peptide uptake in the heart.(6 hours and 24 hours)
- Organ and tissue-specific sensitivity of the 124I-p5+14 Injection radiotracer(6 hours and 24 hours)
- Peptide uptake in the kidney(6 hours and 24 hours)
- Peptide uptake in organ(s) other than kidney or heart if clinically relevant(6 hours and 24 hours)
- Correlation between concentration of the radiotracer (Bq/cc) in the heart with organ-associated clinical biomarkers.(6 hours and 24 hours)
- Correlation between uptake of peptide and clinical status of kidney, heart and other organs(Baseline and 24 hours)