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

NCT01898325 | Unknown DMC

• 1 other identifier: Gaucher_Fibroscan_CTIL
• Study Type: interventional
• Target: 40
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Fibrosis

Keywords

Fibrosis; Gaucher disease

Outcome Measures

Primary Outcomes (1)

• 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.

  2 years

Study Arms (4)

Naïve GD patients

OTHER

Naïve GD patients Intervention: device - Fibroscan

Device: Fibroscan

GD treated with ERT

OTHER

GD treated with ERT Intervention: device - Fibroscan

Device: Fibroscan

Healthy control

OTHER

Healthy control Intervention: device - Fibroscan

Device: Fibroscan

NonAlcoholic Steatohepatitis Patients

OTHER

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

Device: Fibroscan

Interventions

Fibroscan DEVICE

Fibroscan

GD treated with ERT; Healthy control; Naïve GD patients; NonAlcoholic Steatohepatitis Patients

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Active liver disease ,cirrhosis patients.

Sponsors & Collaborators

• Rambam Health Care Campus: lead

MeSH Terms

Conditions

Fibrosis; Gaucher Disease

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms; 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

Study Officials

• Hanna Rosenbaum

  Rambam Health Corporation

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hanna Rosenbaum, MD

CONTACT

972-4-8542541; h_rosenbaum@rambam.health.gov.il

Maher Hijazi

CONTACT

972-50-2064849; drmaher1980@gmail.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2013
• First Posted: July 12, 2013
• Study Start: August 1, 2013
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2016
• Last Updated: July 12, 2013

Record last verified: 2013-07