CEUS Evaluation of Hydrocephalus in Neonates and Infants

NCT06693752 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 24-022232
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Hydrocephalus affects up to 2 out of every 500 births and results in long-term disability in up to 78% of those affected. The standard treatment of hydrocephalus is cerebrospinal fluid (CSF) diversion via placement of an invasive ventricular shunt to relieve elevated intracranial pressure (ICP). The clinical decision for CSF diversion is based on the ventricular size and clinical symptoms which are not robust indicators of brain health in neonatal hydrocephalus. The purpose of this study is to assess the safety and feasibility of performing brain contrast-enhanced ultrasound (CEUS) in neonates and infants with diagnosed and/or suspected hydrocephalus.

Conditions

Hydrocephalus in Infants; Hydrocephalus Acquired

Keywords

Ultrasound; Contrast-enhanced ultrasound; Infant hydrocephalus

Outcome Measures

Primary Outcomes (2)

• Feasibility of brain contrast-enhanced ultrasound (CEUS) examinations in infants with hydrocephalus

  Total number and percentage of successfully competed brain CEUS with adequate quality. Sufficient quality to be interpreted by radiologists without significant motion degradation and/or obscuration due to poor acoustic penetration. Visual rating by 2 teams consisting of primary investigator and second radiologist (co-investigator). Each scan will be rated for diagnostic quality and qualitative rating of cranial perfusion. The visual rating scale used will be: 0 (absent flow), 1 (decreased flow), 2 (normal flow), 3 (increased flow).

  Up to 2 years

• Safety of brain contrast-enhanced ultrasound (CEUS) examinations in infants with hydrocephalus

  Total number/percentage of patients who experienced adverse events (if any) will be reported to assess safety. All subjects entered into the study and receiving at least one injection of investigational drug will be included in the safety analysis. Adverse events will be recorded at 1) 60 minutes post-scanning when the monitoring period is completed, and 2) through 48 hours post-scanning, with documentation at 48 hours post-scanning if no adverse event presents until this point.

  Up to 2 years

Secondary Outcomes (6)

• Correlation between contrast-enhanced ultrasound (CEUS) parameters (intensity perfusion) and invasively measured intracranial pressure (ICP) in infants with hydrocephalus

  Up to 2 years

• Correlation between contrast-enhanced ultrasound (CEUS) parameters (particle-tracking velocimetry) and invasively measured intracranial pressure (ICP) in infants with hydrocephalus

  Up to 2 years

• Correlation between contrast-enhanced ultrasound (CEUS) parameters (particle-image velocimetry) and invasively measured intracranial pressure (ICP) in infants with hydrocephalus

  Up to 2 years

• Correlation between contrast-enhanced ultrasound (CEUS) parameters (intensity perfusion) and cerebral ischemia biomarkers (lactate level and lactate/pyruvate ratio in cerebrospinal fluid) in infants with hydrocephalus

  Up to 2 years

• Correlation between contrast-enhanced ultrasound (CEUS) parameters (particle-tracking velocimetry) and cerebral ischemia biomarkers (lactate level and lactate/pyruvate ratio in cerebrospinal fluid) in infants with hydrocephalus

  Up to 2 years

Study Arms (1)

Brain contrast-enhanced ultrasound

EXPERIMENTAL

The study duration per subject will be up to 30 minutes including the time to prepare the contrast agent, perform the pre-contrast imaging and the contrast-enhanced ultrasound (CEUS), as well as the 60-minute monitoring period after the administration of the contrast agent. A second CEUS exam may be performed within 1-2 weeks from the first scan for up to two CEUS exams of 1 hour and 30-minute duration each. Study participation will be complete when the 60-minute monitoring period of the last CEUS performed is complete.

Drug: Sulfur Hexafluoride Lipid Type A Microspheres 25 mg Injection Powder for Suspension [LUMASON]

Interventions

Sulfur Hexafluoride Lipid Type A Microspheres 25 mg Injection Powder for Suspension [LUMASON] DRUG

Injection of Sulfur hexafluoride lipid-type A microspheres contrast agent will be performed via the existing peripheral intravenous line using the FDA-recommended dose of 0.03 mg/kg before performing the contrast-enhanced ultrasound. The weight-based dose of 0.03 mL per kg will be repeated one time during a single examination. Following each injection, an intravenous flush of 0.9% Sodium Chloride is injected.

Brain contrast-enhanced ultrasound

Eligibility Criteria

• Age: 1 Minute - 18 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Males and females younger than 1.5 years old with diagnosed and/or suspected hydrocephalus.
• Post menstrual age of 26 weeks or older.
• Inpatients at the Children's Hospital of Philadelphia.
• Parental/Legally authorized representative permission.

You may not qualify if:

• Medical history of Lumason hypersensitivity.
• Hemodynamic instability as defined by rapid escalation of cardiopulmonary support in the past 12-24 hours, as defined by the clinical care team.
• Respiratory instability as defined by rapid escalation of respiratory support in the past 12-24 hours (Increased fraction of inspired oxygen (FiO2) requirement and/or nitric oxide).

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Interventions

Suspensions

Intervention Hierarchy (Ancestors)

Colloids; Complex Mixtures; Dosage Forms; Pharmaceutical Preparations

Central Study Contacts

Misun Hwang, MD

CONTACT

267-425-7110; hwangm@chop.edu

Maria Mezher, MD

CONTACT

267-425-5821; mezherm@chop.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Sponsor Investigator

Study Record Dates

• First Submitted: November 11, 2024
• First Posted: November 18, 2024
• Study Start: May 5, 2026
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2027
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)