Evaluation of Safety and Efficacy of Lumason in Pediatric Echocardiography

A Multicenter Clinical Evaluation of Safety and Efficacy of Lumason as a Contrast Agent in Pediatric Echocardiography

Safety and Efficacy Study in pediatric subjects aged 9 to 17 years of age with suboptimal LV EBD on non-contrast 2D echocardiography. Imaging modalities that are used throughout the study in pediatric subjects represented those utilized in routine clinical practice in adults.

Study Overview

• Status: Terminated
• Conditions: Heart Disease
• Intervention / Treatment: Drug: sulphur hexafluoride lipid-type A microspheres

Detailed Description

This was a Phase III, multicenter, open-label study that was to be conducted at approximately 7-12 sites in the United States, Canada and Europe in pediatric patients with suboptimal LV EBD on non-contrast 2D transthoracic echocardiography. It was estimated that 92 patients were to be enrolled to provide 73 evaluable patients.

Three cardiologists unaffiliated with enrolling centers (blinded readers), blinded to the patient's identity and clinical profile were to independently evaluate the echocardiograms. The efficacy analysis was primarily based on the blinded reader evaluations.

Imaging modalities that are used throughout the study in pediatric subjects represented those utilized in routine clinical practice in adults.

One of the sites participating in this study was to be asked to consent a subset of patients for additional blood sampling for analysis of SF6 concentration in blood from a total of 6 patients (3 males and 3 females) in the age group 9 up to 12 years of age and 6 patients (3 males and 3 females) in the age of >12 up to and including 17 years.

The current study was designed to assess the efficacy of Lumason-enhanced echocardiography in pediatric patients.

• Study Type: Interventional
• Enrollment (Actual): 13
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Missouri
    • Saint Louis, Missouri, United States, 63110
      • Washington University School of Medicine in Saint Louis
  • Nebraska
    • Omaha, Nebraska, United States, 68114
      • University of Nebraska

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Provide Written informed consent from parent(s) or legal guardian
• Provide assent when required according to local regulations
• Suspected of having cardiac disease or undergoing evaluation of cardiac anatomy for congenital heart disease
• Undergone a previous transthoracic echocardiogram within one month prior to enrollment resulting in suboptimal left ventricular endocardial border defined as ≥ 2 contiguous segments in any given view that cannot be visualized.

Exclusion Criteria:

• Children < 9 years of age
• Previously enrolled in the study
• Administered any other contrast agent either intravascularly or orally within 48 hours of Lumason administration
• Known right-to-left, bidirectional or transient cardiac shunt (ruled out with agitated saline study performed before administration of Lumason)
• Known hypersensitivity to one or more of the ingredients of the investigational product
• Received an investigational compound within 30 days before enrolling into this study
• Pregnant or lactating female
• Determined by investigator to be unsuitable for the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Lumason; All patients were administered, Lumason (sulphur hexafluoride lipid-type A microspheres) an ultrasound contrast agent as a single 0.03 mL/kg bolus injection during echocardiography.	Drug: sulphur hexafluoride lipid-type A microspheres; Ultrasound imaging contrast agent; Other Names: Lumason; SonoVue

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Total Left Ventricular Endocardial Border Delineation (LV EBD) Scores	The total LV EBD score for a subject was the total of the EBD scores (Inadequate - 0, sufficient - 1, Good - 2) from the 17 segments (from the apical 4-chamber, apical 2-chamber and apical long (3-chamber) views, according to the guidelines of the American Society of Echocardiography). The total LV EBD scores could range from 0 to 34 and are presented below for each of the three readers for both unenhanced ultrasound (UEUS) and contrast-enhanced ultrasound (CEUS). Change from baseline is the difference between UEUS and CEUS in total LV EBD scores.	Immediately post dose-Day 1
Percentage of Participants With Adequate Left Ventricular Opacification (LVO)	LVO was graded as 0 to +3, 4-point rating scale. Left ventricular opacification rating of +2 (non-homogeneous) or +3 (complete and homogeneous) was considered as adequate LVO.	Immediately post dose-Day 1

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Adverse Events	To obtain safety data in pediatric subjects administered Lumason for LV EBD improvement during echocardiography	Up to 72 hours post dose

Collaborators and Investigators

• Sponsor: Bracco Diagnostics, Inc
• Investigators: Study Director: Melda Dolan, M.D., Bracco Diagnostics, Inc

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2015
• Primary Completion (Actual): May 31, 2018
• Study Completion (Actual): August 1, 2018

Study Registration Dates

• First Submitted: October 30, 2014
• First Submitted That Met QC Criteria: October 31, 2014
• First Posted (Estimate): November 4, 2014

Study Record Updates

• Last Update Posted (Actual): July 13, 2020
• Last Update Submitted That Met QC Criteria: June 23, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Heart Diseases
• Other Study ID Numbers: BR1-140

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No