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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified July 2013 by Rambam Health Care Campus
Sponsor:
Information provided by (Responsible Party):
Rambam Health Care Campus
ClinicalTrials.gov Identifier:
NCT01898325
First received: March 11, 2013
Last updated: July 9, 2013
Last verified: July 2013
  Purpose

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.


Condition Intervention
Fibrosis
Device: Fibroscan

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Diagnostic
Official Title: Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan

Resource links provided by NLM:


Further study details as provided by Rambam Health Care Campus:

Primary Outcome Measures:
  • Liver and spleen fibrosis [ Time Frame: 2 years ] [ Designated as safety issue: No ]

    Fibrosis will be estimated by the degree of elastography measured by the liver and spleen stifness in kilopascals (kPa).

    Transient elastography will be performed using a Fibro Scan device (EchoSens, Paris, France). A median value [expressed in kilopascals (kPa)] of 10 successful acquisitions will be s considered the representative measurement of liver stiffness. We consider 10 acquisitions with a success rate of at least 60% and an interquartile range (IQR) lower than 20% as representative measurements.

    The FibroScan® and its dedicated probes make up an active, non-implantable medical device using ultrasound. Designed to rapidly measure liver stiffness in a painless and totally non-invasive manner.

    The FibroScan® is based on the one-dimensional pulse elastography technique.



Estimated Enrollment: 40
Study Start Date: August 2013
Estimated Study Completion Date: December 2016
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Naïve GD patients

Naïve GD patients

Intervention: device - Fibroscan

Device: Fibroscan
Fibroscan
GD treated with ERT
GD treated with ERT Intervention: device - Fibroscan
Device: Fibroscan
Fibroscan
Healthy control
Healthy control Intervention: device - Fibroscan
Device: Fibroscan
Fibroscan
NonAlcoholic Steatohepatitis Patients
Patients with NonAlcoholic Steatohepatitis( NASH) Intervention: device - Fibroscan
Device: Fibroscan
Fibroscan

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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01898325

Contacts
Contact: Hanna Rosenbaum, MD 972-4-8542541 h_rosenbaum@rambam.health.gov.il
Contact: Maher Hijazi 972-50-2064849 drmaher1980@gmail.com

Sponsors and Collaborators
Rambam Health Care Campus
Investigators
Principal Investigator: Hanna Rosenbaum Rambam Health Corporation
  More Information

No publications provided

Responsible Party: Rambam Health Care Campus
ClinicalTrials.gov Identifier: NCT01898325     History of Changes
Other Study ID Numbers: Gaucher_Fibroscan_CTIL
Study First Received: March 11, 2013
Last Updated: July 9, 2013
Health Authority: Israel: Ministry of Health

Keywords provided by Rambam Health Care Campus:
Fibrosis
Gaucher disease

Additional relevant MeSH terms:
Gaucher Disease
Fibrosis
Pathologic Processes
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on August 21, 2014