Study to Determine Safety and Dosage of OPTISON in Pediatric Participants

NCT03740997 | Completed Phase 4 Results FDA Drug

• 1 other identifier: GE-191-008
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 8

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

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)

Outcome Measures

Primary Outcomes (1)

• 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)

  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.

  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 (12)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  0- 72 hours

• 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 Heart Rate

  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

Study Arms (2)

Body Weight Group <=40 kg: OPTISON

EXPERIMENTAL

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.

Drug: Optison

Body Weight Group >40 kg: OPTISON

EXPERIMENTAL

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.

Drug: Optison

Interventions

Optison DRUG

Optison is administered intravenously

Also known as: Perflutren Microspheres Injectable Suspension, USP

Body Weight Group <=40 kg: OPTISON; Body Weight Group >40 kg: OPTISON

Eligibility Criteria

• Age: 9 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• GE Healthcare: lead
• Laboratory Corporation of America: collaborator

Study Sites (8)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

MCG Health, Inc.

Augusta, Georgia, 30912, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States

Location

Cohen Children's Medical Center of New York

Lake Success, New York, 11042, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

MeSH Terms

Interventions

FS 069

Results Point of Contact

• Title: David Thompson, MD, PhD
• Organization: GE HealthCare

david.thompson1@gehealthcare.com

+44 7775 543206

Study Officials

• David Thompson, MD, PhD

  GE Healthcare

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 31, 2018
• First Posted: November 14, 2018
• Study Start: December 1, 2020
• Primary Completion: March 30, 2023
• Study Completion: March 30, 2023
• Last Updated: May 17, 2024
• Results First Posted: May 17, 2024

Record last verified: 2024-03

Locations

US (8)