Study to Determine Dosage of OPTISON in Pediatric Patients

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

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

Study Overview

• Status: Completed
• Conditions: Study to Determine Dosage of OPTISON in Children Between ≥9 and <18 Years of Age Weighing ≥20 kg
• Intervention / Treatment: Drug: Optison

• Study Type: Interventional
• Enrollment (Actual): 37
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Michelle Straszacker; Phone Number: +44 (0) 1491 44 3173; Email: Michelle.Straszacker@ge.com
• Study Contact Backup: Name: MJ Kilkenny; Phone Number: 978-493-7719; Email: MJ.Kilkenny@ge.com

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Children's Hospital
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
  • Georgia
    • Augusta, Georgia, United States, 30912
      • MCG Health, Inc.
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins University School of Medicine
  • New York
    • Lake Success, New York, United States, 11042
      • Cohen Children's Medical Center of New York
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Foundation
  • Texas
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Patients with body weight ≥20 to ≤28 kg; 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; Optison is administered intravenously; Other Names: Perflutren Microspheres Injectable Suspension, USP
Experimental: Patients with body weight >28 to ≤40 kg; 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; Optison is administered intravenously; Other Names: Perflutren Microspheres Injectable Suspension, USP
Experimental: Patients with body weight >40 kg; 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; Optison is administered intravenously; Other Names: Perflutren Microspheres Injectable Suspension, USP

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Time Frame
Comparison of diagnostic confidence of LV EBD and wall motion between non-contrast and OPTISON-enhanced echocardiography at various doses.	72 hours
Comparison of diagnostic confidence in the evaluation of LVEF between non-contrast and OPTISON-enhanced echocardiography.	72 hours

Collaborators and Investigators

• Sponsor: GE Healthcare
• Collaborators: Laboratory Corporation of America
• Investigators: Study Director: Francois Tranquart, M.D., Ph.D., GE Healthcare

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2020
• Primary Completion (Actual): March 30, 2023
• Study Completion (Actual): March 30, 2023

Study Registration Dates

• First Submitted: October 31, 2018
• First Submitted That Met QC Criteria: November 13, 2018
• First Posted (Actual): November 14, 2018

Study Record Updates

• Last Update Posted (Actual): December 18, 2023
• Last Update Submitted That Met QC Criteria: December 15, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Child; Left ventricular ejection fraction (LVEF); Contrast-enhanced echocardiography (CE-ECHO); Transthoracic echocardiogram; Left ventricular opacification (LVO); Left ventricular endocardial border delineation (LV EBD)
• Additional Relevant MeSH Terms: Body Weight
• Other Study ID Numbers: GE-191-008

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No