Study to Determine Dosage of OPTISON in Pediatric Patients

Study Overview
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Brief Summary
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Study to determine the safety and appropriate dosage of OPTISON in pediatric patients since OPTISON has been tested in adult patients only during the clinical development

Official Title
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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

Conditions
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Study to Determine Dosage of OPTISON in Children Between ≥9 and <18 Years of Age Weighing ≥20 kg

Intervention / Treatment
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* Drug: Optison


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The subject is between ≥9 and <18 years of age and weighs ≥20 kg. The subject is clinically indicated to undergo a transthoracic echocardiogram. The subject has a suboptimal non-contrast echocardiogram defined as ≥2 contiguous segments in any given view that cannot be visualized. The subject is able to comply with study procedures. A parent or legal guardian of the subject has signed and dated an informed consent form. Exclusion Criteria: The subject was previously enrolled in this study. The subject has 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 subject has a known or suspected hypersensitivity to any of the components of OPTISON, blood, blood products, or albumin. The subject has pulmonary hypertension or unstable cardiopulmonary conditions. The subject has severe liver disease based on medical history. The subject had a recent (<6 months) neurological event. The subject presents 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 subject or other reason that in the judgment of the investigator makes the subject unsuitable for participation in the study.

Ages Eligible for Study
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Minimum Age: 9 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patients with body weight ≥20 to ≤28 kg<br>In children weighing ≥20 to ≤28 kg, 0.1 or 0.2 mL of OPTISON per injection will be given in ascending order. The cumulative dose will not exceed 1.0 mL. | Drug: Optison<br>* Optison is administered intravenously<br>* Other names: Perflutren Microspheres Injectable Suspension, USP;|
| Experimental: Patients with body weight >28 to ≤40 kg<br>If children weigh >28-≤40 kg, 0.2 or 0.3 mL of OPTISON per injection will be administered in ascending order with total dose not to exceed 1.5 mL. | Drug: Optison<br>* Optison is administered intravenously<br>* Other names: Perflutren Microspheres Injectable Suspension, USP;|
| Experimental: Patients with body weight >40 kg<br>For children whose weight is >40 kg, 0.2 or 0.4 mL of OPTISON per injection will be administered in ascending order with total dose not to exceed 1.8 mL. | Drug: Optison<br>* Optison is administered intravenously<br>* Other names: Perflutren Microspheres Injectable Suspension, USP;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Visualization of the 12 segments of the left ventricle wall in standard apical 4-chamber and 2-chamber views measured by the qualitative endocardial border delineation (EBD) visualization scale. | A qualitative score will be assigned to each left ventricle (LV) segment: 0 = no visualization of the LV endocardial border = poor visualization = fair visualization = good/optimal visualization | 72 hours | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall safety profile in terms of occurrence of adverse events (AEs) following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of changes in measurements of systolic and diastolic blood pressure following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of changes in heart rate following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of changes in respiratory rate following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of changes in pulse oximetry following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of arterial oxygen saturation (SaO2) following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of physical examinations following administration of OPTISON. |  | 72 hours | 
| Overall safety profile in terms of 12-lead electrocardiograms (ECGs) following administration of OPTISON. | Descriptive statistics will be used to describe the observed values and change from baseline for ECG intervals (heart rate, PR, RR, QRS, QT, QTcB [Bazett], QTcF [Fridericia]). | 72 hours | 
| Left ventricular opacification (LVO) assessed by visual peak contrast intensity following intravenous administration of OPTISON at various doses. |  | 72 hours | 
| Left ventricular opacification (LVO) assessed by peak LV contrast filling following intravenous administration of OPTISON at various doses. |  | 72 hours | 
| Duration of contrast enhancement following intravenous administration of OPTISON at various doses. |  | 72 hours | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Contrast-enhanced echocardiography (CE-ECHO), Transthoracic echocardiogram, Child, Left ventricular opacification (LVO), Left ventricular ejection fraction (LVEF), Left ventricular endocardial border delineation (LV EBD)