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FDA agreed to early termination due to difficulties enrolling patients.
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lumason | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| sulphur hexafluoride lipid-type A microspheres | Drug | Ultrasound imaging contrast agent |
|
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Melda Dolan, M.D. | Bracco Diagnostics, Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Washington University School of Medicine in Saint Louis | St Louis | Missouri | 63110 | United States | ||
| University of Nebraska |
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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.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | All subjects who received IP and had data available for both unenhanced ultrasound (UEUS) and contrast-enhanced ultrasound (CEUS) were included in ITD population. | Posted | Mean | 95% Confidence Interval | score on a scale | Immediately post dose-Day 1 |
|
Subjects were monitored for any untoward medical events from the time of signed Informed Consent through 72 hours after IP administration.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA (18.1) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Melda S. Dolan, MD, FACC, FASE, Head, Medical Affairs and Cardiac Ultrasound Programs | Bracco Diagnostics Inc. | 1-609-514-2506 | Melda.Dolan@diag.bracco.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Oct 20, 2014 | May 29, 2020 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D006331 | Heart Diseases |
| ID | Term |
|---|---|
| D002318 | Cardiovascular Diseases |
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| ID | Term |
|---|---|
| C420843 | contrast agent BR1 |
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| Omaha |
| Nebraska |
| 68114 |
| United States |
| years |
|
| Age, Customized | Count of Participants | Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Number | participants |
|
| Height | Mean | Standard Deviation | cm |
|
| Weight | Mean | Standard Deviation | kg |
|
| Body Mass Index | Mean | Standard Deviation | kg/m^2 |
|
Off-site Reader 1 UEUS Assessment
| OG001 | Off-site Reader 1 CEUS | Off-site Reader 1 CEUS Assessment |
| OG002 | Off-site Reader 2 UEUS | Off-site Reader 2 UEUS Assessment |
| OG003 | Off-site Reader 2 CEUS | Off-site Reader 2 CEUS Assessment |
| OG004 | Off-site Reader 3 UEUS | Off-site Reader 3 UEUS Assessment |
| OG005 | Off-site Reader 3 CEUS | Off-site Reader 3 CEUS Assessment |
|
|
| Primary | 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. | All subjects who received IP and had data available for contrast-enhanced ultrasound (CEUS) were included in ITD population. | Posted | Number | 95% Confidence Interval | percentage of participants | Immediately post dose-Day 1 |
|
|
|
| Secondary | Number of Participants With Adverse Events | To obtain safety data in pediatric subjects administered Lumason for LV EBD improvement during echocardiography | Summary of adverse events in the safety population | Posted | Count of Participants | Participants | No | Up to 72 hours post dose |
|
|
|
| 0 |
| 12 |
| 0 |
| 12 |
| 5 |
| 12 |
| Injection site bruising | General disorders | MedDRA (18.1) | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA (18.1) | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA (18.1) | Systematic Assessment |
|
| Abnormal behavior | Psychiatric disorders | MedDRA (18.1) | Systematic Assessment |
|
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (18.1) | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (18.1) | Systematic Assessment |
|
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| Title | Measurements |
|---|---|
|
| Number of Subjects With AEs by Intensity -Severe |
|