Sickle Cell DiseaseNCT02007746
Liver Fibrosis in Sickle Cell Disease
An observational study of Sickle Cell Disease, run by University of Miami at 1 site in United States.
Trial summary
Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions. Read more about this study
- Who is it for?
- Ages 10 to 65 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Liver transient elastography (Fibroscan), Ferriscan and Liver biopsy in iron overloaded subjects.
- Type of treatment
- Surgery or procedure
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2012to 1 Jun 2017
- 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 10 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 30 subjects with sickle cell disease and iron overload. 30 control subjects without history of chronic transfusions (less than 4 transfusions in lifetime) and without evidence of liver pathology.
Who may be able to join
- pediatric patients age 10 years and older with sickle cell disease
- meeting other criteria:
- history of chronic transfusion and iron overload and/or
- known liver disease related to sickle cell or iron overload
- signed consent and assent (as applicable)
- Patients 10 years and older with sickle cell disease without history of chronic transfusions (less than 4 transfusions in a lifetime)
- and without obvious liver disease.
Who cannot join
- children younger than 10 years
- Pregnant females
- Prisoners
- Other causes of liver disease, unrelated to sickle cell or iron overload
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Assessment of Liver Fibrosis in Patients With Sickle Cell Disease
Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions. The purpose of this study is to investigate FibroScan readings in patients with sickle cell disease and iron overload secondary to blood transfusions, and to correlate the FibroScan results with Ferriscan. A comparison with the results of FibroScan to patients with Sickle cell without known liver disease, who have never been on chronic transfusions and with normal liver function profiles will also be made.The primary hypothesis is that the results of FibroScan will correlate with the results of Ferriscan and liver biopsy.
Read the detailed description
Liver biopsy is the gold standard to examine the liver for iron deposits and histology. However, liver biopsy is invasive and involves a risk of bleeding and pain. Biopsy may also miss significant pathology if the small biopsy specimen is taken from an uninvolved part of the liver. Non-invasive techniques such as MRI are now used to evaluate the liver iron content. MRI can visualize the whole liver and measure liver iron content. MRI, however, will not detect liver scarring.
Liver transient elastography (FibroScan) is a non-invasive ultrasound tool for assessing liver fibrosis or scarring by measuring liver stiffness (LSM). Compared with liver biopsy, FibroScan provides immediate results and is a painless, short (3 mins), simple procedure to perform. In some studies FibroScan reports have correlated well with liver biopsy results of fibrosis and cirrhosis, and with Ferriscan, ferritin and liver function tests.
This purpose of this study is to investigate the role of FibroScan in individuals with sickle cell anemia and iron overload or who have a diagnosis of liver disease, and to compare FibroScan readings with magnetic resonance imaging.
We will also compare the results of the Fibroscan with liver biopsy.
This study will also compare the results of FibroScan to patients with Sickle cell without any known liver disease, who have never been on chronic transfusions (defined as no more than 4 transfusions in lifetime) and who have normal liver function profiles.
- Conditions
- Sickle Cell Disease
- Being tested
- Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.
- Keywords
- sickle cell disease, hemochromatosis, liver fibrosis, liver cirrhosis, FibroScan, Ferriscan, liver iron
- Medical subject headings
- Anemia, Sickle Cell, Hemochromatosis, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liver transient elastography (Fibroscan)Other
Also called FibroScan
Fibroscan will be performed as a measure of liver stiffness. The study will be obtained free of charge in this study.
FerriscanOther
Ferriscan will be done to assess the quantity of liver iron in iron overloaded subjects.
Liver biopsy in iron overloaded subjects.Procedure or surgery
Liver biopsy will be done and the results will be compared to Fibroscan results.
| Study group | Role | What they receive |
|---|---|---|
| Subjects with sickle cell disease and iron overload | Patients will have blood tests done to evaluate liver function and general health, then have FibroScan and Ferriscan. A blinded (no access to laboratory parameters or available data) radiologist will interpret the Ferriscan. Liver biopsy will be also obtained (if not done within the previous year). Otherwise, the results of the recent liver biopsy will be collected. Receives: Other: Liver transient elastography (Fibroscan); Other: Ferriscan; Procedure: Liver biopsy in iron overloaded subjects. | |
| control patients with sickle cell disease | Patients 10 years and older with sickle cell disease without history of chronic transfusions (less than 4 transfusions in a lifetime) and without obvious liver disease. Will have liver function blood tests and general health check ups. Then will have FibroScan performed. No liver biopsy will be performed in control patients. Receives: Other: Liver transient elastography (Fibroscan) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Liver transient elastography (FibroScan) of liver iron content and stiffness
Liver transient elastography (FibroScan) uses a probe consisting of an ultrasound transducer located at the end of a vibrating piston. The piston produces a vibration of low amplitude and frequency, which generate a shear wave that passes through the skin and liver tissue. The ultrasound then detects the propagation of the shear wave through the liver (at a depth of 25 - 65 mm below the skin surface) by measuring its velocity. The shear wave velocity is directly related to the tissue stiffness, with a higher velocity equating to higher tissue stiffness, corresponding to increasing severity of fibrosis.
Measured: at imaging visit (3 minutes)
Secondary outcomes (2)
magnetic resonance imaging (MRI) measure of liver iron content and stiffness
Measured: at imaging visit (about 30-60 minutes)
liver function tests (ALT, AST, serum alkaline phosphate, GGTP, total bilirubin, direct bilirubin), complete blood count, platelets, reticulocyte count, serum ferritin to assess liver function and evaluate overall health
Measured: at clinic visit blood draw (about 1 minute)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2013 (estimated)
- Study startJul 2012
- Primary completion1 Jun 2017
- Study completion1 Jun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by University of MiamiThe sponsor's other registered studies.
- 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
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ofelia Alvarez, University of Miami
- Investigators
- Ofelia Alvarez, MD, Principal investigator, University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02007746
- Sponsor's protocol ID
- 20120222
- Status last verified
- May 2021
- First submitted
- 6 Dec 2013
- Last updated
- 26 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Voskaridou E, Plata E, Douskou M, Papadakis M, Delaki EE, Christoulas D, Terpos E. Treatment with deferasirox (Exjade) effectively decreases iron burden in patients with thalassaemia intermedia: results of a pilot study. Br J Haematol. 2010 Jan;148(2):332-4. doi: 10.1111/j.1365-2141.2009.07930.x. Epub 2009 Oct 26. No abstract available.
PubMed 19863539Fraquelli M, Cassinerio E, Roghi A, Rigamonti C, Casazza G, Colombo M, Massironi S, Conte D, Cappellini MD. Transient elastography in the assessment of liver fibrosis in adult thalassemia patients. Am J Hematol. 2010 Aug;85(8):564-8. doi: 10.1002/ajh.21752.
PubMed 20658587
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Liver Fibrosis in Patients With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT02007746
- Acronym / sponsor ID
- 20120222
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, hemochromatosis, liver fibrosis, liver cirrhosis, FibroScan, Ferriscan, liver iron
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Study start
- Jul 2012 (actual)
- Primary completion
- 1 Jun 2017 (actual)
- Study completion
- 1 Jun 2017 (actual)
- First posted
- 11 Dec 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.
Patients with sickle cell disease many have a number of systemic complications, including liver problems. Some of these liver problems lead to liver fibrosis/cirrhosis, secondary to chronic blood transfusions. The purpose of this study is to investigate FibroScan readings in patients with sickle cell disease and iron overload secondary to blood transfusions, and to correlate the FibroScan results with Ferriscan. A comparison with the results of FibroScan to patients with Sickle cell without known liver disease, who have never been on chronic transfusions and with normal liver function profiles will also be made.The primary hypothesis is that the results of FibroScan will correlate with the results of Ferriscan and liver biopsy.
Detailed description
Liver biopsy is the gold standard to examine the liver for iron deposits and histology. However, liver biopsy is invasive and involves a risk of bleeding and pain. Biopsy may also miss significant pathology if the small biopsy specimen is taken from an uninvolved part of the liver. Non-invasive techniques such as MRI are now used to evaluate the liver iron content. MRI can visualize the whole liver and measure liver iron content. MRI, however, will not detect liver scarring.
Liver transient elastography (FibroScan) is a non-invasive ultrasound tool for assessing liver fibrosis or scarring by measuring liver stiffness (LSM). Compared with liver biopsy, FibroScan provides immediate results and is a painless, short (3 mins), simple procedure to perform. In some studies FibroScan reports have correlated well with liver biopsy results of fibrosis and cirrhosis, and with Ferriscan, ferritin and liver function tests.
This purpose of this study is to investigate the role of FibroScan in individuals with sickle cell anemia and iron overload or who have a diagnosis of liver disease, and to compare FibroScan readings with magnetic resonance imaging.
We will also compare the results of the Fibroscan with liver biopsy.
This study will also compare the results of FibroScan to patients with Sickle cell without any known liver disease, who have never been on chronic transfusions (defined as no more than 4 transfusions in lifetime) and who have normal liver function profiles.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Subjects with sickle cell disease and iron overload | – | Other: Liver transient elastography (Fibroscan), Other: Ferriscan, Procedure: Liver biopsy in iron overloaded subjects.Patients will have blood tests done to evaluate liver function and general health, then have FibroScan and Ferriscan. A blinded (no access to laboratory parameters or available data) radiologist will interpret the Ferriscan. Liver biopsy will be also obtained (if not done within the previous year). Otherwise, the results of the recent liver biopsy will be collected. |
| control patients with sickle cell disease | – | Other: Liver transient elastography (Fibroscan)Patients 10 years and older with sickle cell disease without history of chronic transfusions (less than 4 transfusions in a lifetime) and without obvious liver disease. Will have liver function blood tests and general health check ups. Then will have FibroScan performed. No liver biopsy will be performed in control patients. |
Who may qualify
- Ages
- 10 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- 30 subjects with sickle cell disease and iron overload. 30 control subjects without history of chronic transfusions (less than 4 transfusions in lifetime) and without evidence of liver pathology.
Key inclusion criteria (7 listed)
- pediatric patients age 10 years and older with sickle cell disease
- meeting other criteria:
- signed consent and assent (as applicable)
- Patients 10 years and older with sickle cell disease without history of chronic transfusions (less than 4 transfusions in a lifetime)
- and without obvious liver disease.
Key exclusion criteria (4 listed)
- children younger than 10 years
- Pregnant females
- Prisoners
- Other causes of liver disease, unrelated to sickle cell or iron overload
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 |
|---|---|---|
| Liver transient elastography (Fibroscan)Also: FibroScanFibroscan will be performed as a measure of liver stiffness. The study will be obtained free of charge in this study. | Other | Subjects with sickle cell disease and iron overload, control patients with sickle cell disease |
| FerriscanFerriscan will be done to assess the quantity of liver iron in iron overloaded subjects. | Other | Subjects with sickle cell disease and iron overload |
| Liver biopsy in iron overloaded subjects.Liver biopsy will be done and the results will be compared to Fibroscan results. | Procedure or surgery | Subjects with sickle cell disease and iron overload |
Primary outcome
Liver transient elastography (FibroScan) of liver iron content and stiffness
Time frame: at imaging visit (3 minutes)
Liver transient elastography (FibroScan) uses a probe consisting of an ultrasound transducer located at the end of a vibrating piston. The piston produces a vibration of low amplitude and frequency, which generate a shear wave that passes through the skin and liver tissue. The ultrasound then detects the propagation of the shear wave through the liver (at a depth of 25 - 65 mm below the skin surface) by measuring its velocity. The shear wave velocity is directly related to the tissue stiffness, with a higher velocity equating to higher tissue stiffness, corresponding to increasing severity of fibrosis.
2 secondary outcomes
magnetic resonance imaging (MRI) measure of liver iron content and stiffness
Time frame: at imaging visit (about 30-60 minutes)
liver function tests (ALT, AST, serum alkaline phosphate, GGTP, total bilirubin, direct bilirubin), complete blood count, platelets, reticulocyte count, serum ferritin to assess liver function and evaluate overall health
Time frame: at clinic visit blood draw (about 1 minute)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2021.
- Results publication: PubMed 19863539Voskaridou E, Plata E, Douskou M, Papadakis M, Delaki EE, Christoulas D, Terpos E. Treatment with deferasirox (Exjade) effectively decreases iron burden in patients with thalassaemia intermedia: results of a pilot study. Br J Haematol. 2010 Jan;148(2):332-4. doi: 10.1111/j.1365-2141.2009.07930.x. Epub 2009 Oct 26. No abstract available.
- Results publication: PubMed 20658587Fraquelli M, Cassinerio E, Roghi A, Rigamonti C, Casazza G, Colombo M, Massironi S, Conte D, Cappellini MD. Transient elastography in the assessment of liver fibrosis in adult thalassemia patients. Am J Hematol. 2010 Aug;85(8):564-8. doi: 10.1002/ajh.21752.
- Individual participant dataSharing planned: No
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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.
Ofelia Alvarez, MD principal investigator
University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology
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 Sickle Cell Disease, run by University of Miami at 1 site in United States.
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 7 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: finished, as of the sponsor's last update on 26 May 2021. 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 United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Liver transient elastography (FibroScan) of liver iron content and stiffness (time frame: at imaging visit (3 minutes)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02007746, which also lists 2 linked publications.
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 26 May 2021.