Gaucher DiseaseNCT03896607
Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography
An observational study of Gaucher Disease and Liver Fibrosis, run by HaEmek Medical Center, Israel at 1 site in Israel.
Trial summary
The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher Disease(GD) by using Shear Wave Elastography- SWE to evaluation fibrosis of the tissue and to check the correlation of fibrosis with the biomarkers of disease severity. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gaucher Disease, Liver Fibrosis, Spleen; Fibrosis
- How many people
- 18 participants
- Where
- 1 siteIsrael
- Study dates
- 30 Mar 2019to 31 Oct 2022
- Phase
- Observational
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: adult patient with Gaucher disease.
Who may be able to join
- Patients with Gaucher disease over the age of 18 years.
- Patients able to sign informed consent form.
- Women are not pregnant.
- Patients who read and understood the contents of the consent form and gave their written consent to participate in the study.
Who cannot join
- Participants under the age of 18.
- Pregnant women.
- Participants are unable to sign an informed consent form.
- Participants with known liver disease not associated with Gaucher disease such as liver cirrhosis, patients with HBV, HBC or liver cirrhosis.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Prospective Study: Shear-Wave Elastography of the Liver and the Spleen in Adult Patients With Gaucher's Disease
The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher Disease(GD) by using Shear Wave Elastography- SWE to evaluation fibrosis of the tissue and to check the correlation of fibrosis with the biomarkers of disease severity.
Read the detailed description
To assess liver and spleen stiffness measurement using Shear Wave Elastography-SWE and evaluate liver and spleen fibrosis in patients with Gaucher Disease. To compare the elastography with the biomarkers that taken at the GD haematology clinic and to check the correlation with severity of liver and spleen fibrosis.
SWE 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 relatively new and safe method could avoid complications and invasive procedure in GD patients .
- Conditions
- Gaucher Disease, Liver Fibrosis, Spleen; Fibrosis
- Keywords
- shear wave elastography
- Medical subject headings
- Gaucher Disease, Liver Cirrhosis, Fibrosis
What is being measured
The primary outcome is the main question the study is designed to answer.
Shear Wave Elastography of liver to asses liver fibrosis in Gaucher disease patients.
Shear wave elastography(SWE),an ultrasound imaging technique that can produce 2D and 3D images and quantified measurements of tissue stiffness noninvasively. SWE obtained from the right liver lobe for staging liver fibrosis according to Metavir Fibrosis Score with sensitivity86%-98% and specificity 90%-93%. SWE well corelated with Metavir Fibrosis Score which is system used to assess the extent of inflammation and fibrosis by histopathological evaluation in a liver biopsy. The biomarkers of disease severity will be correlated to METAVIR score. METAVIR score(F0-F4) ,Fibrosis Level measured by KPa(kiloPascal) using SWE. F0 = Below 5 KPa, No fibrosis. F1=5.0-7.1 KPa, Mild Fibrosis. F2=7.1-8.7 KPa, Significant Fibrosis. F3=8.7-10.4 KPa, Sever Fibrosis. F4=10.4-19 KPa ,Cirrhosis. SWE can differentiate patients with no or minimal (F0 and F1) fibrosis from those with severe fibrosis or cirrhosis (F3 and F4) with no need for biopsy unless there are other factors that need to be considered.
Measured: 0-2 hours
Secondary outcomes (1)
Shear Wave Elastography of spleen to asses spleen fibrosis in Gaucher disease patients.
Shear wave elastography (SWE), an ultrasound imaging technique that can produce 2D and 3D images and quantified measurements of tissue stiffness noninvasively.
Participants with Gaucher disease will under go shear wave elastography for spleen.
Ultrasound Elastography is a non-invasive test which uses the technology of Shear Wave Elastography (SWE) obtained from the spleen for staging tissue fibrosis according to a study, Spleen Elastography was performed in 59 healthy individuals and the mean value measured in KPa (kiloPascal) was 16.6 ± 2.5 kPa, independent of patient age, sex or spleen size.
above the normal value it is considerd fibrosis of the tisuue of the spleen.
Measured: 0-2 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2019
- Study start30 Mar 2019
- Primary completion31 Oct 2022
- Study completion31 Oct 2022
Why it stopped: study has ended due to internal changes
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- HaEmek Medical Center, Israel
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ziv Neeman, HaEmek Medical Center, Israel
- Investigators
- ziv neeman, MD, Principal investigator, haemek medical center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03896607
- Sponsor's protocol ID
- 0135-18-EMC
- Status last verified
- Mar 2023
- First submitted
- 17 Mar 2019
- Last updated
- 6 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Study: Shear-Wave Elastography of the Liver and the Spleen in Adult Patients With Gaucher's Disease
- ClinicalTrials.gov ID
- NCT03896607
- Acronym / sponsor ID
- 0135-18-EMC
- Conditions
- Gaucher Disease, Liver Fibrosis, Spleen; Fibrosis
- Keywords
- shear wave elastography
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: study has ended due to internal changes
- Enrolment
- 18 participants (actual)
- Lead sponsor
- HaEmek Medical Center, IsraelUniversity, hospital or other
- Study start
- 30 Mar 2019 (actual)
- Primary completion
- 31 Oct 2022 (actual)
- Study completion
- 31 Oct 2022 (actual)
- First posted
- 1 Apr 2019 (actual)
- 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(GD) by using Shear Wave Elastography- SWE to evaluation fibrosis of the tissue and to check the correlation of fibrosis with the biomarkers of disease severity.
Detailed description
To assess liver and spleen stiffness measurement using Shear Wave Elastography-SWE and evaluate liver and spleen fibrosis in patients with Gaucher Disease. To compare the elastography with the biomarkers that taken at the GD haematology clinic and to check the correlation with severity of liver and spleen fibrosis.
SWE 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 relatively new and safe method could avoid complications and invasive procedure in GD patients .
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- adult patient with Gaucher disease.
Key inclusion criteria (4 listed)
- Patients with Gaucher disease over the age of 18 years.
- Patients able to sign informed consent form.
- Women are not pregnant.
- Patients who read and understood the contents of the consent form and gave their written consent to participate in the study.
Key exclusion criteria (4 listed)
- Participants under the age of 18.
- Pregnant women.
- Participants are unable to sign an informed consent form.
- Participants with known liver disease not associated with Gaucher disease such as liver cirrhosis, patients with HBV, HBC or liver cirrhosis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Shear Wave Elastography of liver to asses liver fibrosis in Gaucher disease patients.
Time frame: 0-2 hours
Shear wave elastography(SWE),an ultrasound imaging technique that can produce 2D and 3D images and quantified measurements of tissue stiffness noninvasively. SWE obtained from the right liver lobe for staging liver fibrosis according to Metavir Fibrosis Score with sensitivity86%-98% and specificity 90%-93%. SWE well corelated with Metavir Fibrosis Score which is system used to assess the extent of inflammation and fibrosis by histopathological evaluation in a liver biopsy. The biomarkers of disease severity will be correlated to METAVIR score. METAVIR score(F0-F4) ,Fibrosis Level measured by KPa(kiloPascal) using SWE. F0 = Below 5 KPa, No fibrosis. F1=5.0-7.1 KPa, Mild Fibrosis. F2=7.1-8.7 KPa, Significant Fibrosis. F3=8.7-10.4 KPa, Sever Fibrosis. F4=10.4-19 KPa ,Cirrhosis. SWE can differentiate patients with no or minimal (F0 and F1) fibrosis from those with severe fibrosis or cirrhosis (F3 and F4) with no need for biopsy unless there are other factors that need to be considered.
1 secondary outcome
Shear Wave Elastography of spleen to asses spleen fibrosis in Gaucher disease patients.
Time frame: 0-2 hours
Results and safety
The study was stopped early (reason given: study has ended due to internal changes). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2023.
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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.
ziv neeman, MD principal investigator
haemek medical center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Gaucher Disease and Liver Fibrosis, run by HaEmek Medical Center, Israel at 1 site in Israel.
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 4 inclusion and 4 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: stopped early, as of the sponsor's last update on 6 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
The study was stopped early (reason given: study has ended due to internal changes). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Shear Wave Elastography of liver to asses liver fibrosis in Gaucher disease patients. (time frame: 0-2 hours). 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 6 Mar 2023.