Noninvasive Subharmonic Aided Pressure Estimation of Portal Hypertension in Children

Who is this study for? Patients with Portal Hypertension
What treatments are being studied? Subharmonic Aided Pressure Estimation Measurement using the Ultrasound Contrast Agent
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

Early diagnosis of portal hypertension is difficult as symptoms rarely manifest until the later stages of liver disease. Both cirrhotic and non-cirrhotic portal hypertension can result in life-threatening complications, the most frequent of which is bleeding from esophageal varices. In children, variceal bleeds are associated with mortality rates of 1-3 %, while life-threatening complications have been reported in up to 20 % of children with cirrhosis. Despite the high incidence of portal hypertension in children with liver disease, a noninvasive modality to monitor disease progression and risk of complications is currently lacking. Hence, this trial will investigate the safety and efficacy of subharmonic aided pressure estimation (SHAPE) as a noninvasive ultrasound technique for diagnosing portal hypertension in children.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 21
Healthy Volunteers: f
View:

• Patients with a diagnosis of chronic liver disease without portal hypertension.

• Patients with a diagnosis of chronic liver disease with portal hypertension.

Locations
United States
Pennsylvania
The Children's Hospital of Philadelphia
RECRUITING
Philadelphia
Contact Information
Primary
Morgan L Gabbert, MA
gabbertm@email.chop.edu
267-426-0820
Time Frame
Start Date: 2021-11-03
Estimated Completion Date: 2025-08
Participants
Target number of participants: 120
Treatments
Experimental: SHAPE with Sonazoid
Up to 60 children (6-21 years of age) with a diagnosis of chronic liver disease, including some who will have portal hypertension who are followed at The Children's Hospital of Philadelphia (CHOP) will be enrolled in this arm. A SHAPE measurement using the ultrasound contrast agent Sonazoid® (perfluorobutane microbubbles) will be performed during a single visit. A dose of three vials with 16 μL each of microbubbles will be prepared. An infusion of the ultrasound contrast agent at the rate of 0.18 mL/kg/hour will be co-infused with an infusion of saline at the rate of 120 mL/hour resulting in an effective dosage of 1.44 μL microbubbles/kg/hour as per the FDA approved IND through an IV line in a peripheral vein. The total duration of contrast agent infusion is expected to range from 4 to 8 minutes, which includes a 2 min calibration period followed by 2-6 minutes of SHAPE acquisition (i.e., ultrasound imaging).
Active_comparator: SHAPE with Lumason
Up to 60 children (6-21 years of age) with a diagnosis of chronic liver disease, including some who will have portal hypertension who are followed at CHOP will be enrolled in this arm. A SHAPE measurement using the ultrasound contrast agent LUMASON® (sulfur hexafluoride lipid-type A microspheres) will be performed during a single visit. Two doses of 0.03 mL/kg (or 2.4 mL maximum) as per package labeling will be prepared and mixed with saline at a 1:10 dilution in a 50 ml bag of saline. The diluted preparation of Lumason will be through an IV line in a peripheral vein up to 4 mL/min using an infusion setup. The total duration of contrast agent infusion is expected to range from 4 to 8 minutes, which includes a 2 min calibration period followed by 2-6 minutes of SHAPE acquisition (i.e., ultrasound imaging).
Experimental: Longitudinal SHAPE
SHAPE will be used to monitor subjects identified in the initial examination as having portal hypertension for up to 18 months. These subjects will undergo laboratory testing every 6 months as a part of their clinical standard of care. During these times the SHAPE examination will also be repeated using the same ultrasound contrast agent and infusion methodologies (including dosages) as during the initial study.
Sponsors
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Children's Hospital of Philadelphia
Leads: Thomas Jefferson University

This content was sourced from clinicaltrials.gov