PERFLUBUTANE for Focal liver lesion

Inclusion criteria

• {"criterion_text":"- The subject is <18 years of age on the day of consent."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- The subject has had a dynamic CECT or CEMRI examination within the past month for malignant lesions, or 3 months for benign lesions if performed retrospectively, or is scheduled to have one in the month following inclusion in the study, and these images are/would be available."}
• {"criterion_text":"- The subject can comply with study procedures."}
• {"criterion_text":"- 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"}
• {"criterion_text":"- Post-menarcheal female patients must have a negative urine pregnancy test at screening and at pre-dose on the dosing day."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- The subject has a body weight of 3 kg or greater."}
• {"criterion_text":"- The subject has a negative egg allergy test if the egg allergy status of the subject is unknown."}

Exclusion criteria

• {"criterion_text":"- 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), or a positive result from an egg-allergy test performed at screening. Note: If the egg-allergy status is unknown, an egg-allergy test must be performed at screening and the result must be negative."}
• {"criterion_text":"- The subject has an acute clinically fatal condition (i.e., the expected survival is ≤6 months)."}
• {"criterion_text":"- The subject has previously received Sonazoid™ or another ultrasound contrast agent within the past 30 days."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- The subject has undergone or was undergoing systemic or loco-regional chemotherapy or radiation therapy."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- The subject is a pregnant or lactating female."}
• {"criterion_text":"- The physician judges that a large-enough needle (24-gauge or larger) cannot be inserted."}
• {"criterion_text":"- The subject has a known right-left cardiac shunt."}

Primary endpoints

• {"endpoint_text":"- The accuracy, sensitivity and 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.","definition_or_measurement_approach":"Diagnostic accuracy, sensitivity and specificity calculated from independent blinded reads of Sonazoid™-enhanced vascular-phase images versus the subject’s baseline diagnosis established by CECT, CEMRI or biopsy; WFUMB-EFSUMB guidelines used for image interpretation."}

Secondary endpoints

• {"endpoint_text":"- Accuracy, sensitivity and specificity of the diagnosis (benign or malignant FLL) based on the Sonazoid™-enhanced imaging by vascular phase imaging, as compared with the unenhanced ultrasound imaging, when the baseline diagnosis is used as the reference standard.","definition_or_measurement_approach":"Compare diagnostic metrics (accuracy, sensitivity, specificity) from Sonazoid™-enhanced vascular-phase imaging to unenhanced ultrasound using baseline diagnosis (CECT/CEMRI/biopsy) as reference."}
• {"endpoint_text":"- Accuracy, sensitivity and specificity of the diagnosis (benign or malignant FLL) based on the Sonazoid™-enhanced imaging by vascular phase and Kupffer phase imaging, as compared with the unenhanced ultrasound imaging, when the baseline diagnosis is used as the reference standard.","definition_or_measurement_approach":"Compare diagnostic metrics from combined vascular and Kupffer phase Sonazoid™-enhanced imaging to unenhanced ultrasound using baseline diagnosis as reference."}
• {"endpoint_text":"- The difference in the diagnostic confidence score for the unenhanced ultrasound imaging and that for the Sonazoid™-enhanced ultrasound imaging based on the vascular phase.","definition_or_measurement_approach":"Measure and compare reader diagnostic confidence scores between unenhanced ultrasound and Sonazoid™-enhanced vascular-phase images."}
• {"endpoint_text":"- Exploratory outcome measure: The difference in the diagnostic confidence score for the unenhanced ultrasound imaging and that for the Sonazoid™-enhanced ultrasound imaging based on the vascular and Kupffer phase.","definition_or_measurement_approach":"Exploratory comparison of reader confidence scores between unenhanced and Sonazoid™-enhanced images across vascular and Kupffer phases."}
• {"endpoint_text":"- Exploratory outcome measure: CEUS specific FLL diagnosis throughout the vascular phase of imaging and the vascular and Kupffer phases of imaging according to WFUMB-EFSUMB guidelines (HCC, metastasis, haemangioma, FNH, focal fatty infiltration, focal fatty sparing) observed in paediatric patients.","definition_or_measurement_approach":"Descriptive categorisation of CEUS-specific focal liver lesion diagnoses observed in Sonazoid™-enhanced vascular and Kupffer phase imaging per WFUMB-EFSUMB guidelines."}
• {"endpoint_text":"- Safety outcome measure: Treatment-emergent AEs (TEAEs) defined as AEs that occurred at any timepoint from the administration of Sonazoid™ to the end of the study (72 hours after Sonazoid™ administration), including drug-related TEAEs","definition_or_measurement_approach":"Record and summarise all AEs occurring from administration to 72 hours post-administration; classify as TEAEs and report drug-related TEAEs."}
• {"endpoint_text":"- Safety outcome measure: Serious AEs (SAEs)","definition_or_measurement_approach":"Record and report serious adverse events according to standard SAE definitions and timelines."}
• {"endpoint_text":"- Safety outcome measure: Physical examination, vital signs, blood and urine samples for clinical laboratory testing and injection site monitoring","definition_or_measurement_approach":"Collect and report clinical laboratory tests, vital signs, physical exam findings and injection site assessments as safety measures."}

Other endpoints

• {"endpoint_text":"- Exploratory outcome measure: The difference in the diagnostic confidence score for the unenhanced ultrasound imaging and that for the Sonazoid™-enhanced ultrasound imaging based on the vascular and Kupffer phase.","definition_or_measurement_approach":"Exploratory comparison of reader confidence scores between unenhanced and Sonazoid™-enhanced images across vascular and Kupffer phases."}
• {"endpoint_text":"- Exploratory outcome measure: CEUS specific FLL diagnosis throughout the vascular phase of imaging and the vascular and Kupffer phases of imaging according to WFUMB-EFSUMB guidelines (HCC, metastasis, haemangioma, FNH, focal fatty infiltration, focal fatty sparing) observed in paediatric patients.","definition_or_measurement_approach":"Descriptive categorisation of CEUS-specific focal liver lesion diagnoses observed in Sonazoid™-enhanced vascular and Kupffer phase imaging per WFUMB-EFSUMB guidelines."}

Germany

Latest Decision Or Authorization Date

06-11-2025

Number Of Sites

1

Number Of Participants

10

Italy

Latest Decision Or Authorization Date

04-11-2025

Number Of Sites

2

Number Of Participants

20

Primary sponsor

Full Name

GE Healthcare Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

United Kingdom

Contract research organisations

Name

PPD Development LP

Responsibilities

Codes: 1,10,5,6

Name

Medidata Solutions Inc.

Responsibilities

Codes: 7

Third parties

• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"Codes: 1,10,5,6","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Codes: 7","organisation_type":"Non-Pharmaceutical company"}