Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan

NCT ID: NCT01898325

Last Updated: 2013-07-12

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: NA
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2013-08-31
• Study Completion Date: 2016-12-31

Brief Summary

The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher disease divided into two groups, naive GD patients and GD patients treated with ERT. As liver biopsy in these patients not recommended because the risk of bleeding using Fibroscan is a safe with diagnostic accuracy regarding the liver (& Spleen) fibrosis. Estimating spleen fibrosis is an innovative approach in liver disease and Gaucher.

The evaluation of fibrosis with this new and safe method could avoid complications antiinvasive procedure in GD patients. The addition of fibrosis biomarkers will help for patients score evaluation. The finding of liver and spleen stiffness will be evaluated in native and ERT treated Gaucher patients in order to assess ERT effect on fibrosis.

The Aims are: 1) To assess liver and spleen stiffness measurement using fibroscan and evaluate liver and spleen fibrosis in patients with GD.

2) To compare the elastography in two cohorts of GD patients: ERT treated and naïve GD patients and two control groups of patients: healthy and Non Alcoholic Steatohepatitis (NASH) patients.

3) To correlate the elastography findings with clinical and laboratory data in the four patient groups focusing on Gaucher disease manifestations and GD severity. To compare the elastography in GD naïve and ERT treated patients.

Detailed Description

The investigated cohort will include 4 groups of patients 20 probands in each group including: 1) Naïve GD patients 2) GD treated with ERT 3) Healthy control 4) Patients with NonAlcoholic Steatohepatitis( NASH) which are followed at hepatology unit of the Ziv Hospital and healthy controls.

Serum fibrosis markers will be tested including: Bilirubin ,GGT, Haptoglobin,AST,ALT .The findings of spleen and liver fibrosis will be correlated with disease severity usingZimran's Severity Score Index (SSI) liver function tests , serology for viral hepatitis, GDbiomarkers, hemoglobin, platelet levels, and a GD severity score.

Protein C will be measured in all groups of patients Protein C activity as it may be used as a sensitive marker of hepatocellular damage even in those patients with mild liver affection .Also Patients with cirrhosis possess an imbalance in pro-coagulant versus anticoagulantactivity due to increased factor VIII and decreased protein C.

Conditions

Fibrosis

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: DIAGNOSTIC
• Blinding Strategy: NONE

Study Groups

Naïve GD patients

Naïve GD patients

Intervention: device - Fibroscan

Group Type: OTHER

Fibroscan

Intervention Type: DEVICE

Fibroscan

GD treated with ERT

GD treated with ERT Intervention: device - Fibroscan

Group Type: OTHER

Fibroscan

Intervention Type: DEVICE

Fibroscan

Healthy control

Healthy control Intervention: device - Fibroscan

Group Type: OTHER

Fibroscan

Intervention Type: DEVICE

Fibroscan

NonAlcoholic Steatohepatitis Patients

Patients with NonAlcoholic Steatohepatitis( NASH) Intervention: device - Fibroscan

Group Type: OTHER

Fibroscan

Intervention Type: DEVICE

Fibroscan

Interventions

Fibroscan

Fibroscan

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

• Diagnosis of Gaucher in treatment and without treatment.
• Healthy control.
• Patients with Non Alcoholic Steatohepatitis( NASH) which are followed at hepatology unit.

Exclusion Criteria

• Active liver disease ,cirrhosis patients.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Rambam Health Care Campus

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Hanna Rosenbaum

Role: PRINCIPAL_INVESTIGATOR

Rambam Health Corporation

Central Contacts

Hanna Rosenbaum, MD

Role: CONTACT

h_rosenbaum@rambam.health.gov.il

972-4-8542541

Maher Hijazi

Role: CONTACT

drmaher1980@gmail.com

972-50-2064849

Other Identifiers

Gaucher_Fibroscan_CTIL

Identifier Type: -

Identifier Source: org_study_id