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