Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

NCT03896607 | Terminated

• 1 other identifier: 0135-18-EMC
• Study Type: observational
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher Disease(GD) by using Shear Wave Elastography- SWE to evaluation fibrosis of the tissue and to check the correlation of fibrosis with the biomarkers of disease severity.

Conditions

Gaucher Disease; Liver Fibrosis; Spleen; Fibrosis

Keywords

shear wave elastography

Outcome Measures

Primary Outcomes (1)

• Shear Wave Elastography of liver to asses liver fibrosis in Gaucher disease patients.

  Shear wave elastography(SWE),an ultrasound imaging technique that can produce 2D and 3D images and quantified measurements of tissue stiffness noninvasively. SWE obtained from the right liver lobe for staging liver fibrosis according to Metavir Fibrosis Score with sensitivity86%-98% and specificity 90%-93%. SWE well corelated with Metavir Fibrosis Score which is system used to assess the extent of inflammation and fibrosis by histopathological evaluation in a liver biopsy. The biomarkers of disease severity will be correlated to METAVIR score. METAVIR score(F0-F4) ,Fibrosis Level measured by KPa(kiloPascal) using SWE. F0 = Below 5 KPa, No fibrosis. F1=5.0-7.1 KPa, Mild Fibrosis. F2=7.1-8.7 KPa, Significant Fibrosis. F3=8.7-10.4 KPa, Sever Fibrosis. F4=10.4-19 KPa ,Cirrhosis. SWE can differentiate patients with no or minimal (F0 and F1) fibrosis from those with severe fibrosis or cirrhosis (F3 and F4) with no need for biopsy unless there are other factors that need to be considered.

  0-2 hours

Secondary Outcomes (1)

• Shear Wave Elastography of spleen to asses spleen fibrosis in Gaucher disease patients.

  0-2 hours

Other Outcomes (1)

• Biomarkers of the Gaucher Disease

  Up to 1 month

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

adult patient with Gaucher disease.

You may qualify if:

• Patients with Gaucher disease over the age of 18 years.
• Patients able to sign informed consent form.
• Women are not pregnant.
• Patients who read and understood the contents of the consent form and gave their written consent to participate in the study.

You may not qualify if:

• Participants under the age of 18.
• Pregnant women.
• Participants are unable to sign an informed consent form.
• Participants with known liver disease not associated with Gaucher disease such as liver cirrhosis, patients with HBV, HBC or liver cirrhosis.

Sponsors & Collaborators

• HaEmek Medical Center, Israel: lead

Study Sites (1)

HaEmek Medical Center

Afula, Northern District, 1834111, Israel

Location

MeSH Terms

Conditions

Gaucher Disease; Liver Cirrhosis; Fibrosis

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Liver Diseases; Digestive System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• ziv neeman, MD

  haemek medical center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Radiology department

Study Record Dates

• First Submitted: March 17, 2019
• First Posted: April 1, 2019
• Study Start: March 30, 2019
• Primary Completion: October 31, 2022
• Study Completion: October 31, 2022
• Last Updated: March 6, 2023

Record last verified: 2023-03

Locations

IL (1)