FibrosisNCT01898325
Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan
A study testing Fibroscan in people with Fibrosis, run by Rambam Health Care Campus.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Fibrosis
- What is being tested?
- Fibroscan
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- No sites listed yet
- Study dates
- Aug 2013to Dec 2016
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Diagnosis of Gaucher in treatment and without treatment.
- Healthy control.
- Patients with Non Alcoholic Steatohepatitis( NASH) which are followed at hepatology unit.
Who cannot join
- Active liver disease ,cirrhosis patients.
Trial locations and contacts
No sites are listed yet.
About this study
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.
Read more
The Aims are: 1) To assess liver and spleen stiffness measurement using fibroscan and evaluate liver and spleen fibrosis in patients with GD.
- 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.
- 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.
Read the 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
- Being tested
- Fibroscan
- Keywords
- Fibrosis, Gaucher disease
- Medical subject headings
- Fibrosis, Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FibroscanDevice
Fibroscan
| Study group | Role | What they receive |
|---|---|---|
| Naïve GD patients | Other | Naïve GD patients
Intervention: device - Fibroscan Receives: Device: Fibroscan |
| GD treated with ERT | Other | GD treated with ERT Intervention: device - Fibroscan Receives: Device: Fibroscan |
| Healthy control | Other | Healthy control Intervention: device - Fibroscan Receives: Device: Fibroscan |
| NonAlcoholic Steatohepatitis Patients | Other | Patients with NonAlcoholic Steatohepatitis( NASH) Intervention: device - Fibroscan Receives: Device: Fibroscan |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2013 (estimated)
- Study startAug 2013
- Primary completionDec 2015 (estimated)
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rambam Health Care Campus
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hanna Rosenbaum, Principal investigator, Rambam Health Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01898325
- Sponsor's protocol ID
- Gaucher_Fibroscan_CTIL
- Status last verified
- Jul 2013
- First submitted
- 11 Mar 2013
- Last updated
- 12 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan
- ClinicalTrials.gov ID
- NCT01898325
- Acronym / sponsor ID
- Gaucher_Fibroscan_CTIL
- Conditions
- Fibrosis
- Keywords
- Fibrosis, Gaucher disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Rambam Health Care CampusUniversity, hospital or other
- Study start
- Aug 2013
- Primary completion
- Dec 2015 (estimated)
- Study completion
- Dec 2016 (estimated)
- First posted
- 12 Jul 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
- 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.
- 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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Naïve GD patients | Other | Device: FibroscanNaïve GD patients Intervention: device - Fibroscan |
| GD treated with ERT | Other | Device: FibroscanGD treated with ERT Intervention: device - Fibroscan |
| Healthy control | Other | Device: FibroscanHealthy control Intervention: device - Fibroscan |
| NonAlcoholic Steatohepatitis Patients | Other | Device: FibroscanPatients with NonAlcoholic Steatohepatitis( NASH) Intervention: device - Fibroscan |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of Gaucher in treatment and without treatment.
- Healthy control.
- Patients with Non Alcoholic Steatohepatitis( NASH) which are followed at hepatology unit.
Key exclusion criteria (1 listed)
- Active liver disease ,cirrhosis patients.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FibroscanFibroscan | Device | GD treated with ERT, Healthy control, Naïve GD patients, NonAlcoholic Steatohepatitis Patients |
Primary outcome
Liver and spleen fibrosis
Time frame: 2 years
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jul 2013.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Hanna Rosenbaum principal investigator
Rambam Health Corporation
Questions about this trial
What is this clinical trial studying?
A study testing Fibroscan in people with Fibrosis, run by Rambam Health Care Campus.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hanna Rosenbaum, MD (972-4-8542541, h_rosenbaum@rambam.health.gov.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Liver and spleen fibrosis (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jul 2013.