Study to Determine Safety and Dosage of OPTISON in Pediatric Participants

General Electric (GE)

Status and phase: Completed; Phase 4

Conditions: Transthoracic Echocardiography; Suspected or Known Structural or Functional Cardiac Abnormality

Treatments: Drug: Optison

Study type: Interventional

Funder types: Industry

Identifiers

NCT03740997

GE-191-008

Details and patient eligibility

About

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

Enrollment: 39 patients

Sex: All

Ages: 9 to 17 years old

Volunteers: No Healthy Volunteers

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).

Trial design

Primary purpose: Diagnostic

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

39 participants in 2 patient groups

Body Weight Group <=40 kg: OPTISON

Experimental group

Description:

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.

Treatment:

Drug: Optison

Body Weight Group >40 kg: OPTISON

Experimental group

Description:

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.

Treatment:

Drug: Optison