Clinical trial NCT03740997: Study to Determine Safety and Dosage of OPTISON in Pediatric Participants
Official title: 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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Transthoracic Echocardiography
Suspected or Known Structural or Functional Cardiac Abnormality
Keywords: Contrast-enhanced echocardiography (CE-ECHO)
Transthoracic echocardiogram
Child
Left ventricular opacification (LVO)
Left ventricular ejection fraction (LVEF)
Left ventricular endocardial border delineation (LV EBD)
Lead sponsor: GE Healthcare
Sponsor class: INDUSTRY
Collaborators: name: Laboratory Corporation of America
First posted: 2018-11-14
Enrollment: 39
Sex: ALL
Minimum age (years): 9
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: 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


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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).

Primary outcomes:
measure: 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)
timeFrame: 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
Secondary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: 0- 72 hours
measure: Change From Baseline in Systolic and Diastolic Blood Pressure
timeFrame: Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1
measure: Change From Baseline in Heart Rate
timeFrame: Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1
measure: Change From Baseline in Respiratory Rate
timeFrame: Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1
measure: Change From Baseline in Oxygen Saturation as Measured by Pulse Oximetry
timeFrame: Baseline (pre-dose), and at 10 and 60 minutes post last dose on Day 1
measure: Number of Participants With Clinically Significant Abnormality in Physical Examination Findings
timeFrame: From first dose (Day 1) of study drug to 60 minutes post last dose on Day 1
measure: Change From Baseline in 12-lead Electrocardiograms (ECGs) Parameters
timeFrame: Baseline (pre-dose), and at 10 and 30 minutes post last dose on Day 1
measure: Number of Participants by Degree of Left Ventricular Opacification (LVO) Assessed by Visual Peak Contrast Intensity
timeFrame: 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
measure: Number of Participants by Grade of Left Ventricular Opacification (LVO) Assessed by Peak Left Ventricular (LV) Contrast Filling
timeFrame: 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
measure: Number of Participants by Contrast Enhancement Duration in the Left Ventricular (LV) Chamber
timeFrame: 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
measure: Number of Participants by Diagnostic Confidence of Left Ventricular Endocardial Border Delineation (LV EBD) and Wall Motion for Contrast and Non-Contrast Enhanced Images
timeFrame: 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
measure: Number of Participants by Diagnostic Confidence of Left Ventricular Ejection Fraction (LVEF) for Non-contrast and OPTISON Enhanced Echocardiography
timeFrame: 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
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Augusta
country: United States
facility: MCG Health, Inc.
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
city: Lake Success
country: United States
facility: Cohen Children's Medical Center of New York
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
Study officials:
affiliation: GE Healthcare
name: David Thompson, MD, PhD
role: STUDY_DIRECTOR
References: