SHAPE of Portal Hypertension in Children

NCT04720456 · Thomas Jefferson University

Recruiting

In plain English

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Official title

Noninvasive Subharmonic Aided Pressure Estimation of Portal Hypertension in Children

About this study

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 criteria

Inclusion Criteria: * Patients with a diagnosis of chronic liver disease without portal hypertension. * Patients with a diagnosis of chronic liver disease with portal hypertension. Exclusion Criteria: * Subjects who are pregnant. * Patients with known or suspected hypersensitivity to egg phosphatidyl serine or with a history of anaphylactic allergy to eggs or egg products. * Subjects with allergy to egg products or other components of the ultrasound contrast agents will be excluded. * History of allergic reaction to Lumason®, sulfur hexafluoride, sulfur hexafluoride lipid microsphere components, or other ingredients in Lumason (polyethylene glycol, distearoylphosphatidylcholine (DSPC), dipalmitoylphosphatidylglycerol sodium (DPPG-Na), palmitic acid) * History of allergic reaction to Sonazoid * Patients with biliary atresia with asplenia or polysplenia. * Patients with prior liver transplant. * Patients with cystic fibrosis. * Patients with chronic lung disease. * Patients with portal vein thrombosis, cavernous transformation of the portal vein or absent portal vein. * Adults not competent/impaired. * Patients with significant heart disease or severe congenital heart disease

Study design

Enrollment target: 120 participants

Allocation: randomized

Masking: none

Age groups: child, adult

Timeline

Starts: 2021-11-03

Estimated completion: 2025-08

Last updated: 2025-02-11

Interventions

Drug: SHAPE measurement using the ultrasound contrast agent Sonazoid (perfluorobutane microbubbles)Drug: SHAPE measurement using the ultrasound contrast agent Lumason (sulfur hexafluoride lipid-type A microspheres)

Primary outcomes

• The rate (%) of adverse events that occur with Sonazoid compared to the current rate of adverse events that have been reported in the Lumason package insert for pediatric use (0.001%) (2 hours)
• Calibrated subharmonic microbubble signals (in dB) between the portal and hepatic veins will differentiate between the portal hypertension and non-portal hypertension groups with an accuracy of 94% (2 hours)

Sponsor

Thomas Jefferson University · other

With: Children's Hospital of Philadelphia, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Contacts & investigators

ContactMorgan L Gabbert, MA · contact · gabbertm@email.chop.edu · 267-426-0820

InvestigatorFlemming Forsberg, PhD · principal_investigator, Thomas Jefferson University

InvestigatorSudha A Anupindi, MD · principal_investigator, Children's Hospital of Philadelphia

All locations (1)

The Children's Hospital of PhiladelphiaRecruiting

Philadelphia, Pennsylvania, United States

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