Phase 4 Paediatric Study to Evaluate Sonazoid Safety and Efficacy for Contrast-Enhanced Ultrasound Liver Imaging

NCT06639828 | Recruiting Phase 4

• 3 other identifiers: GE-045-40110074502023-505569-95-00
• Study Type: interventional
• Target: 50
• Locations: 3 countries
• Sites: 6

Brief Summary

Liver tumours, 40% of which are benign, account for 1% to 4% of all solid tumours in children. The benign tumours are mainly haemangiomas, liver hamartomas, and liver cell adenomas. The malignant tumours are mainly hepatoblastoma, hepatocellular carcinoma (HCC), malignant liver mesothelioma, and rhabdomyosarcoma. The differential diagnosis of liver masses in children is generally based on the child's age, the clinical evaluation (including alpha-fetoprotein test results), and imaging characteristics. Liver tumours seldom produce clear symptoms and signs in the early stages, and they progress rapidly. As a result, most liver tumours are not diagnosed until the middle or late stages. To improve survival among patients with liver tumours, clinicians must find the liver tumours as early as possible and determine whether the tumours are benign or malignant. Conventional ultrasonography is commonly used for screening, and is preferred as the first-line imaging technique for children. Not only does it have a wide range of applications and a high diagnostic yield, but it is well accepted by patients and their parents. It can be performed in the examination room with the parents present, allowing real-time imaging and direct interaction with patient and parents. CEUS is a non-invasive imaging technology that can continuously and dynamically observe blood perfusion in tumours in real-time through injection of a contrast agent to enhance the blood flow reflux signal in the human body. Consequently, CEUS is useful for visualising capillaries and tissue perfusion in the human body. The purpose of this current study is to evaluate the use of Sonazoid™ perfluorobutane (PFB) microbubbles as a contrast medium for CEUS for evaluation of solitary liver masses in children.

Conditions

Focal Liver Lesions

Keywords

Contrast-Enhanced Ultrasound

Outcome Measures

Primary Outcomes (3)

• Accuracy of Sonazoid-enhanced ultrasound at imaging focal liver lesions during the vascular phase of imaging

  Accuracy of the diagnosis based on the independent blinded read of the Sonazoid™-enhanced imaging for lesion differentiation (benign or malignant FLLs) during vascular phase imaging. The WFUMB-EFSUMB guidelines for interpreting CEUS of the liver in adults shall be used for interpreting the images and the subject's baseline diagnosis (well-established by CECT, CEMRI or biopsy) will be used as the reference diagnosis/standard of truth.

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 4-minutes post injection for the vascular phase] and blinded image evaluations shall be executed remotely at a central lab

• Sensitivity of Sonazoid-enhanced ultrasound at imaging focal liver lesions during the vascular phase of imaging

  Sensitivity of the diagnosis based on the independent blinded read of the Sonazoid™-enhanced imaging for lesion differentiation (benign or malignant FLLs) during vascular phase imaging. The WFUMB-EFSUMB guidelines for interpreting CEUS of the liver in adults shall be used for interpreting the images and the subject's baseline diagnosis (well-established by CECT, CEMRI or biopsy) will be used as the reference diagnosis/standard of truth.

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 4-minutes post injection for the vascular phase] and blinded image evaluations shall be executed remotely at a central lab

• Specificity of Sonazoid-enhanced ultrasound at imaging focal liver lesions during the vascular phase of imaging

  Specificity of the diagnosis based on the independent blinded read of the Sonazoid™-enhanced imaging for lesion differentiation (benign or malignant FLLs) during vascular phase imaging. The WFUMB-EFSUMB guidelines for interpreting CEUS of the liver in adults shall be used for interpreting the images and the subject's baseline diagnosis (well-established by CECT, CEMRI or biopsy) will be used as the reference diagnosis/standard of truth.

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 4-minutes post injection for the vascular phase] and blinded image evaluations shall be executed remotely at a central lab

Secondary Outcomes (13)

• Comparison of accuracy of Sonazoid-enhanced ultrasound to Unenhanced ultrasound at imaging focal liver lesions during vascular phase

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 4-minutes post injection for the vascular phase] and blinded image evaluations shall be executed remotely at a central lab.

• Comparison of accuracy of Sonazoid-enhanced ultrasound to Unenhanced ultrasound at imaging focal liver lesions during vascular and Kupffer phase

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 10-minutes post injection for the vascular and Kupffer phase(s)] and blinded image evaluations shall be executed remotely at a central lab

• Comparison of sensitivity of Sonazoid-enhanced ultrasound to Unenhanced ultrasound at imaging focal liver lesions during vascular phase

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 4-minutes post injection for the vascular phase] and blinded image evaluations shall be executed remotely at a central lab.

• Comparison of sensitivity of Sonazoid-enhanced ultrasound to Unenhanced ultrasound at imaging focal liver lesions during vascular and Kupffer phase

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 10-minutes post injection for the vascular and Kupffer phase(s)] and blinded image evaluations shall be executed remotely at a central lab

• Comparison of specificity of Sonazoid-enhanced ultrasound to Unenhanced ultrasound at imaging focal liver lesions during vascular phase

  Images to be captured during the studies CEUS examination [approximately 5 seconds prior to bolus injection, up to 4-minutes post injection for the vascular phase] and blinded image evaluations shall be executed remotely at a central lab.

Study Arms (1)

Sonazoid

EXPERIMENTAL

Participants will receive a single iv dose of Sonazoid™, dosed according to body weight (0.12 μL microbubbles/kg)

Diagnostic Test: Contrast-Enhanced Ultrasound

Interventions

Contrast-Enhanced Ultrasound DIAGNOSTIC_TEST

Following unenhanced (pre-contrast) ultrasound imaging of the target focal liver lesions (FLL), Sonazoid™ will be intravenously administered and a contrast-enhanced examination will be performed.

Sonazoid

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• The subject is \<18 years of age on the day of consent.
• The subject has at least 1 untreated FLL with ≤8 lesions (excluding cysts), with ≤8 cm confirmed in a diagnostic examination performed in the past month (or past 3 months if the lesion was benign) and that could also be visualised when obtained via unenhanced ultrasound imaging
• The subject can comply with study procedures.
• Parents or legally authorised representatives have signed the Informed Consent Form approved for this study by the Independent Review Board (IRB)/Independent Ethics Committee (IEC). The form will indicate that the patient (and/or a legally acceptable representative) has been informed of all pertinent aspects of the study. Patients who are able to provide assent have signed an age-appropriate paediatric assent form
• Post-menarcheal female patients must have a negative urine pregnancy test at screening and at pre-dose on the dosing day.
• Post-menarcheal female patients and male patients who are sexually active with a partner of childbearing potential must be practicing abstinence or be using an effective form of birth control (See Section 8.6) for ≥30 days before being enrolled in the study
• The subject has a body weight of 3 kg or greater.
• The subject has a negative egg allergy test if the egg allergy status of the subject is unknown.

You may not qualify if:

• The subject has a known or suspected hypersensitivity to any of the components of Sonazoid™, including a history of allergies to eggs or egg products (i.e., manifested by full body rash, respiratory difficulty, oral or laryngeal swelling, hypotension, or shock).
• The subject has an acute clinically fatal condition (i.e., the expected survival is ≤6 months).
• The subject has previously received Sonazoid™ or another ultrasound contrast agent within the past 30 days.
• The subject had undergone or is planning to undergo an examination with a contrast agent (i.e., iodinated x-ray contrast agent, MRI contrast agent or another ultrasound contrast agent) within the 24 hours before or after Sonazoid™ injection.
• The subject has undergone or was undergoing systemic or loco-regional chemotherapy or radiation therapy
• The subject is participating in another clinical trial with an unregistered medicinal product, or less than 30 days has passed since the subject completed participation in such a trial
• The subject is a pregnant or lactating female.
• The physician judges that a large-enough needle (24-gauge or larger) cannot be inserted.
• The subject has a known right-left cardiac shunt.

Sponsors & Collaborators

• GE Healthcare: lead
• PPD Development, LP: collaborator
• Medidata Solutions: collaborator

Study Sites (6)

Munich University Hospital

München, 81377, Germany

NOT YET RECRUITING

Papa Giovanni XXIII Hospital

Bergamo, 24127, Italy

NOT YET RECRUITING

Giannina Gaslini Institute

Genoa, 16147, Italy

NOT YET RECRUITING

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS2 8BJ, United Kingdom

RECRUITING

King's College Hospital NHS Foundation Trust

London, SE5 9RS, United Kingdom

RECRUITING

Imperial College Healthcare NHS Trust

London, W2 1NY, United Kingdom

RECRUITING

Study Officials

• David Thompson

  GE Healthcare

  STUDY DIRECTOR

Central Study Contacts

Tingting Wang

CONTACT

+86 21 3895 4500; Tingting.Wang12@gehealthcare.com

David Thompson

CONTACT

+44 (0) 7385 117614; david.thompson1@gehealthcare.com

Study Design

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

Study Record Dates

• First Submitted: October 1, 2024
• First Posted: October 15, 2024
• Study Start: January 20, 2025
• Primary Completion (Estimated): July 31, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: March 4, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

IT (2); DE (1); GB (3)