A Phase 4, Open-Label, Non-Randomized, Multicenter Study to Evaluate Safety and Efficacy of Intravenous Administration of OPTISON™ for Contrast- Enhanced Echocardiography in Pediatric Patients
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 39
- 试验地点
- 8
- 主要终点
- Mean Total Visualization Score of Qualitative Endocardial Border Delineation (EBD) of the 12 Segments of the Left Ventricle (LV) Wall in Standard Apical 4-chamber (A4C) and Apical 2-chamber (A2C)
研究概览
简要总结
Study to determine the safety and appropriate dosage of OPTISON in pediatric participants since OPTISON has been tested in adult participants only during the clinical development
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 9 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant was between ≥9 and <18 years of age and weighs ≥20 kg.
- •The participant was clinically indicated to undergo a transthoracic echocardiogram.
- •The participant had a suboptimal non-contrast echocardiogram defined as ≥2 contiguous segments in any given view that cannot be visualized.
- •The participant was able to comply with study procedures.
- •A parent or legal guardian of the participant had signed and dated an informed consent form.
- •Post-menarchal female participants must have had a negative urine pregnancy test at screening and at pre-dose on the day of OPTISON administration.
- •Post-menarchal female participants must have been practicing abstinence, or be using an effective form of birth control (e.g., intrauterine device, oral contraceptives, contraceptive implants or injections, diaphragm with spermicide, cervical cap, or consort use of condom) for at least 30 days before being enrolled in the study
排除标准
- •The participant was previously enrolled in this study.
- •The participant received an investigational medicinal product within 30 days before or is scheduled to receive one from time of entry into this study until completion of the follow-up period proposed for this study.
- •The participant had a known or suspected hypersensitivity to any of the components of OPTISON, blood, blood products, or albumin.
- •The participant had pulmonary hypertension or unstable cardiopulmonary conditions.
- •The participant had severe liver disease based on medical history.
- •The participant had a recent (<6 months) neurological event.
- •The participant presented any clinically active, serious, life-threatening disease, with a life expectancy of less than 1 month or where study participation may compromise the management of the participant or other reason that in the judgment of the investigator makes the participant unsuitable for participation in the study.
- •The participant was a pregnant or lactating female, or was a female of childbearing potential not using an acceptable form of birth control (negative urine pregnancy test was also required).
研究组 & 干预措施
Body Weight Group <=40 kg: OPTISON
Participants with body weight greater than or equal to (>=) 20 to less than or equal to (<=) 28 kilograms (kg) received OPTISON 0.1 milliliter (mL; dose level 1) or 0.2 mL (dose level 2); and participants with body weight greater than (>) 28 to <=40 kg received OPTISON 0.2 mL (dose level 1) or 0.3 mL (dose level 2) intravenous (IV) injection, 10 minutes apart on Day 1. The maximum allowed cumulative dose for body weight >=20 to <=28 kg and >28 to <=40 kg was 1.0 mL and 1.5 mL per dose level, respectively.
The treated subjects received both dose levels. Per protocol, up to 3 injections per dose level were allowed for acquiring different imaging views. A maximum of 2 injections was recorded in the treated population.
干预措施: Optison (Drug)
Body Weight Group >40 kg: OPTISON
Participants with body weight >40 kg received OPTISON 0.2 (dose level 1) or 0.4 mL (dose level 2) IV injection, 10 minutes apart on Day 1. The maximum allowed cumulative dose was 1.8 mL per dose level.
The treated subjects received both dose levels. Per protocol, up to 3 injections per dose level were allowed for acquiring different imaging views. A maximum of 2 injections was recorded in the treated population.
干预措施: Optison (Drug)
结局指标
主要结局
Mean Total Visualization Score of Qualitative Endocardial Border Delineation (EBD) of the 12 Segments of the Left Ventricle (LV) Wall in Standard Apical 4-chamber (A4C) and Apical 2-chamber (A2C)
时间窗: Images were captured during the study echocardiogram (0 to 30 minutes) on Day 1 and blinded image evaluations (BIE) were carried out at the study core laboratory following image transfer
Visualization of each of the 12 segments of the LV wall in standard A4C and A2C views were measured by the qualitative EBD visualization scale: score 0 =no visualization of the LV endocardial border; 1 =poor visualization; 2 =fair visualization; 3 =good/optimal visualization. The total LV EBD score was calculated as the sum of the individual scores assigned to each of the 12 LV wall segment and the total score ranged from 0 (no visualization of the LV endocardial border) to 36 (good/optimal visualization). A higher score indicated better visualization. The total score of qualitative EBD visualization scale were reported by reader (independent blinded), non-contrast and dose levels.
次要结局
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(0- 72 hours)
- Number of Participants by Degree of Left Ventricular Opacification (LVO) Assessed by Visual Peak Contrast Intensity(Images were captured during the study echocardiogram (0 to 30 minutes) on Day 1 and blinded image evaluations (BIE) were carried out at the study core laboratory following image transfer)
- Change From Baseline in Heart Rate(Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1)
- Change From Baseline in 12-lead Electrocardiograms (ECGs) Parameters(Baseline (pre-dose), and at 10 and 30 minutes post last dose on Day 1)
- Number of Participants With Clinically Significant Abnormality in Physical Examination Findings(From first dose (Day 1) of study drug to 60 minutes post last dose on Day 1)
- Number of Participants by Grade of Left Ventricular Opacification (LVO) Assessed by Peak Left Ventricular (LV) Contrast Filling(Images were captured during the study echocardiogram (0 to 30 minutes) on Day 1 and blinded image evaluations (BIE) were carried out at the study core laboratory following image transfer)
- Number of Participants by Diagnostic Confidence of Left Ventricular Endocardial Border Delineation (LV EBD) and Wall Motion for Contrast and Non-Contrast Enhanced Images(Images were captured during the study echocardiogram (0 to 30 minutes) on Day 1 and blinded image evaluations (BIE) were carried out at the study core laboratory following image transfer)
- Change From Baseline in Systolic and Diastolic Blood Pressure(Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1)
- Change From Baseline in Respiratory Rate(Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1)
- Change From Baseline in Oxygen Saturation as Measured by Pulse Oximetry(Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1)
- Number of Participants by Contrast Enhancement Duration in the Left Ventricular (LV) Chamber(Images were captured during the study echocardiogram (0 to 30 minutes) on Day 1 and blinded image evaluations (BIE) were carried out at the study core laboratory following image transfer)
- Number of Participants by Diagnostic Confidence of Left Ventricular Ejection Fraction (LVEF) for Non-contrast and OPTISON Enhanced Echocardiography(Images were captured during the study echocardiogram (0 to 30 minutes) on Day 1 and blinded image evaluations (BIE) were carried out at the study core laboratory following image transfer)