Trial Outcomes & Findings for Evaluation of Safety and Efficacy of Lumason in Pediatric Echocardiography (NCT NCT02282163)
NCT ID: NCT02282163
Last Updated: 2020-07-13
Results Overview
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.
TERMINATED
PHASE3
13 participants
Immediately post dose-Day 1
2020-07-13
Participant Flow
Study Initiation Date (first subject enrolled): 20 October 2015; Study Completion Date (last subject completed study related activities): 20 January 2018. Enrolment was at 2 sites in the US. Even though last subject enrolled was 20 January 2018, FDA release from completion of enrolment was 31 May 2018. Blinded read completion was 13 August 2018.
The study was terminated early due to challenges with enrollment. At the time of termination, 13 subjects were enrolled and 12 subjects were dosed with Lumason, thus the efficacy analysis was considered as exploratory and p-values are not included in the Results section. PK assessment was not performed due to enrollment difficulties.
Participant milestones
| Measure |
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.
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
12
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
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.
|
|---|---|
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Overall Study
Protocol Violation
|
1
|
Baseline Characteristics
Evaluation of Safety and Efficacy of Lumason in Pediatric Echocardiography
Baseline characteristics by cohort
| Measure |
Lumason
n=12 Participants
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.
|
|---|---|
|
Age, Continuous
|
13.8 years
STANDARD_DEVIATION 2.29 • n=99 Participants
|
|
Age, Customized
Age group · 6 to 9 years
|
1 Participants
n=99 Participants
|
|
Age, Customized
Age group · 10 to 11 years
|
0 Participants
n=99 Participants
|
|
Age, Customized
Age group · 12 to 15 years
|
8 Participants
n=99 Participants
|
|
Age, Customized
Age group · 16-17
|
3 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
|
Race/Ethnicity, Customized
White
|
10 participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Black
|
2 participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 participants
n=99 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 participants
n=99 Participants
|
|
Height
|
156.1 cm
STANDARD_DEVIATION 28.51 • n=99 Participants
|
|
Weight
|
60.76 kg
STANDARD_DEVIATION 23.846 • n=99 Participants
|
|
Body Mass Index
|
26.35 kg/m^2
STANDARD_DEVIATION 12.092 • n=99 Participants
|
PRIMARY outcome
Timeframe: Immediately post dose-Day 1Population: All subjects who received IP and had data available for both unenhanced ultrasound (UEUS) and contrast-enhanced ultrasound (CEUS) were included in ITD population.
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.
Outcome measures
| Measure |
Off-site Reader 1 UEUS
n=12 Participants
Off-site Reader 1 UEUS Assessment
|
Off-site Reader 1 CEUS
n=12 Participants
Off-site Reader 1 CEUS Assessment
|
Off-site Reader 2 UEUS
n=12 Participants
Off-site Reader 2 UEUS Assessment
|
Off-site Reader 2 CEUS
n=12 Participants
Off-site Reader 2 CEUS Assessment
|
Off-site Reader 3 UEUS
n=12 Participants
Off-site Reader 3 UEUS Assessment
|
Off-site Reader 3 CEUS
n=12 Participants
Off-site Reader 3 CEUS Assessment
|
|---|---|---|---|---|---|---|
|
Change From Baseline in Total Left Ventricular Endocardial Border Delineation (LV EBD) Scores
|
6.4 score on a scale
Interval 2.9 to 10.0
|
33.5 score on a scale
Interval 32.6 to 34.3
|
10.6 score on a scale
Interval 6.8 to 14.4
|
33.3 score on a scale
Interval 31.8 to 34.9
|
7.3 score on a scale
Interval 4.0 to 10.5
|
33.0 score on a scale
Interval 31.8 to 34.2
|
PRIMARY outcome
Timeframe: Immediately post dose-Day 1Population: All subjects who received IP and had data available for contrast-enhanced ultrasound (CEUS) were included in ITD population.
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.
Outcome measures
| Measure |
Off-site Reader 1 UEUS
n=12 Participants
Off-site Reader 1 UEUS Assessment
|
Off-site Reader 1 CEUS
n=12 Participants
Off-site Reader 1 CEUS Assessment
|
Off-site Reader 2 UEUS
n=12 Participants
Off-site Reader 2 UEUS Assessment
|
Off-site Reader 2 CEUS
Off-site Reader 2 CEUS Assessment
|
Off-site Reader 3 UEUS
Off-site Reader 3 UEUS Assessment
|
Off-site Reader 3 CEUS
Off-site Reader 3 CEUS Assessment
|
|---|---|---|---|---|---|---|
|
Percentage of Participants With Adequate Left Ventricular Opacification (LVO)
|
100 percentage of participants
Interval 73.5 to 100.0
|
100 percentage of participants
Interval 73.5 to 100.0
|
100 percentage of participants
Interval 73.5 to 100.0
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Up to 72 hours post dosePopulation: Summary of adverse events in the safety population
To obtain safety data in pediatric subjects administered Lumason for LV EBD improvement during echocardiography
Outcome measures
| Measure |
Off-site Reader 1 UEUS
n=12 Participants
Off-site Reader 1 UEUS Assessment
|
Off-site Reader 1 CEUS
Off-site Reader 1 CEUS Assessment
|
Off-site Reader 2 UEUS
Off-site Reader 2 UEUS Assessment
|
Off-site Reader 2 CEUS
Off-site Reader 2 CEUS Assessment
|
Off-site Reader 3 UEUS
Off-site Reader 3 UEUS Assessment
|
Off-site Reader 3 CEUS
Off-site Reader 3 CEUS Assessment
|
|---|---|---|---|---|---|---|
|
Number of Participants With Adverse Events
Number of Subjects With at Least 1 AE
|
5 Participants
|
—
|
—
|
—
|
—
|
—
|
|
Number of Participants With Adverse Events
Number of Subjects With AEs by Intensity - Mild
|
4 Participants
|
—
|
—
|
—
|
—
|
—
|
|
Number of Participants With Adverse Events
Number of Subjects With AEs by Intensity -Moderate
|
1 Participants
|
—
|
—
|
—
|
—
|
—
|
|
Number of Participants With Adverse Events
Number of Subjects With AEs by Intensity -Severe
|
0 Participants
|
—
|
—
|
—
|
—
|
—
|
Adverse Events
Lumason
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Lumason
n=12 participants at risk
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.
|
|---|---|
|
Gastrointestinal disorders
Vomiting
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
|
General disorders
Injection site bruising
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
|
General disorders
Pyrexia
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
|
Nervous system disorders
Headache
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
|
Psychiatric disorders
Abnormal behavior
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.3%
1/12 • Number of events 1 • Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
|
Additional Information
Melda S. Dolan, MD, FACC, FASE, Head, Medical Affairs and Cardiac Ultrasound Programs
Bracco Diagnostics Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place