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

Primary Purpose

Fibrosis

Status
Unknown status
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Fibroscan
Sponsored by
Rambam Health Care Campus
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Fibrosis focused on measuring Fibrosis, Gaucher disease

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm 4

    Arm Type

    Other

    Other

    Other

    Other

    Arm Label

    Naïve GD patients

    GD treated with ERT

    Healthy control

    NonAlcoholic Steatohepatitis Patients

    Arm Description

    Naïve GD patients Intervention: device - Fibroscan

    GD treated with ERT Intervention: device - Fibroscan

    Healthy control Intervention: device - Fibroscan

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

    Outcomes

    Primary Outcome Measures

    Liver and spleen fibrosis
    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.

    Secondary Outcome Measures

    Full Information

    First Posted
    March 11, 2013
    Last Updated
    July 9, 2013
    Sponsor
    Rambam Health Care Campus
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01898325
    Brief Title
    Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan
    Official Title
    Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    July 2013
    Overall Recruitment Status
    Unknown status
    Study Start Date
    August 2013 (undefined)
    Primary Completion Date
    December 2015 (Anticipated)
    Study Completion Date
    December 2016 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Rambam Health Care Campus

    4. Oversight

    Data Monitoring Committee
    Yes

    5. Study Description

    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Fibrosis
    Keywords
    Fibrosis, Gaucher disease

    7. Study Design

    Primary Purpose
    Diagnostic
    Study Phase
    Not Applicable
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    40 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Naïve GD patients
    Arm Type
    Other
    Arm Description
    Naïve GD patients Intervention: device - Fibroscan
    Arm Title
    GD treated with ERT
    Arm Type
    Other
    Arm Description
    GD treated with ERT Intervention: device - Fibroscan
    Arm Title
    Healthy control
    Arm Type
    Other
    Arm Description
    Healthy control Intervention: device - Fibroscan
    Arm Title
    NonAlcoholic Steatohepatitis Patients
    Arm Type
    Other
    Arm Description
    Patients with NonAlcoholic Steatohepatitis( NASH) Intervention: device - Fibroscan
    Intervention Type
    Device
    Intervention Name(s)
    Fibroscan
    Intervention Description
    Fibroscan
    Primary Outcome Measure Information:
    Title
    Liver and spleen fibrosis
    Description
    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.
    Time Frame
    2 years

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    80 Years
    Accepts Healthy Volunteers
    Accepts Healthy Volunteers
    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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Hanna Rosenbaum, MD
    Phone
    972-4-8542541
    Email
    h_rosenbaum@rambam.health.gov.il
    First Name & Middle Initial & Last Name or Official Title & Degree
    Maher Hijazi
    Phone
    972-50-2064849
    Email
    drmaher1980@gmail.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Hanna Rosenbaum
    Organizational Affiliation
    Rambam Health Corporation
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

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

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