Hereditary AnemiasNCT02067130
Utility of Fibroscan in Estimating Hepatic Iron Concentration
A study testing Fibroscan in people with Hereditary Anemias, run by University of British Columbia at 1 site in Canada.
Trial summary
In patients with hereditary anemias (e.g. thalassemias), defective red blood cells are produced due to an error in the genes, or DNA, that provide the instructions for their synthesis. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Hereditary Anemias
- What is being tested?
- Fibroscan
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteCanada
- Study dates
- Oct 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 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- All adult patients (age 19 or greater) with hereditary anemias requiring chronic blood transfusion at St. Paul's Hospital will be invited to participate in this study. The majority of patients will be β-Thalassemia Major.
Who cannot join
- Known Hepatitis B positive
- Known Hepatitis C positive
- Known HIV positive
- Known liver cirrhosis
- Known primary liver disease such as Wilson's disease and hereditary hemochromatosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Utility of Transient Elastography (Fibroscan) in Estimating Hepatic Iron Concentration in Comparison to MRI in Patients With Transfusion Dependent Hemoglobinopathies
In patients with hereditary anemias (e.g. thalassemias), defective red blood cells are produced due to an error in the genes, or DNA, that provide the instructions for their synthesis. As a result, hereditary anemias are characterized by chronically low hemoglobin, which is contained inside red blood cells and carries oxygen throughout the body. In more severe cases, patients are dependent on frequent blood transfusions to replenish the hemoglobin.
The body has limited ability to get rid of excess iron. However, with repeated blood transfusions, the iron level in the body builds up because the red blood cells contain iron as heme. Over time, the high level of iron accumulates in organs such as the heart, liver, and pancreas causing heart problems, liver failure, and diabetes. As a result, patients who receive multiple blood transfusions need to be monitored for iron overload, and be started on medical therapy in a timely fashion to prevent organ damage.
Read more
Liver is usually the first and the most affected organ by iron accumulation, so knowledge of its iron concentration provides estimate of total body iron load. Liver biopsy is the gold standard in measuring the iron concentration in the liver, but it is invasive and cannot be performed on routine basis. MRI is another option that can assess liver iron concentration non-invasively, and is currently recommended for monitoring iron load on a yearly basis. However, MRI has a high cost and is not easily accessible in Canada. The investigators aim to determine if transient elastography (Fibroscan), which is a form of ultrasound that measures liver stiffness, can accurately assess liver iron concentration.
Hypothesis:
Fibroscan reading correlates with MRI and serum ferritin in estimating hepatic iron concentration.
Read the detailed description
Inclusion criteria: All adult patients (age 19 or greater) with hereditary anemias requiring chronic blood transfusion at St. Paul's Hospital will be invited to participate in this study. The majority of patients will be β-Thalassemia Major. They will be given a pamphlet containing the details of the study and can contact the research assistant or clinic nurse for more information should they be interested.
Exclusion criteria: Patients with known Hepatitis B positive, known Hepatitis C positive, known HIV positive, known liver cirrhosis, known primary liver disease such as Wilson's disease and hereditary hemochromatosis are excluded from the study.
Written consent will be obtained from all participants by the clinic nurse/research assistant prior to enrollment.
Study procedures:
Data to be collected retrospectively from patient charts (St. Paul's Hospital's EMR/Sunrise Clinical Manager and paper chart) include: baseline demographic data (age, gender, hematological condition), medical comorbidities and complications related to iron overload (Diabetes, hypothyroidism, cardiomyopathy/arrhythmia and congestive heart failure, hypogonadotropic hypogonadism, osteopenia and osteoporosis syndrome..etc), current medications including use of iron chelators such as desferrioxamine, deferasirox, deferiprone or combination therapy, viral hepatitis status (B and C) and date of test, liver cirrhosis status (stage), liver biopsy result (iron concentration) and date of procedure. Patients with chronic transfusion requirement usually undergo annual MRI at the beginning of the year to estimate hepatic iron concentration as per standard practice. This year (2013), R2 MRI (FerriScan) will also be available for the first time to all patients in BC as part of routine monitoring for iron overload. Details/results from both techniques (i.e. same images collected in one scan, but analyzed differently using R2 and T2* algorithms) will be collected.
- Conditions
- Hereditary Anemias
- Being tested
- Fibroscan
- Keywords
- Beta Thalassemia Major
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FibroscanProcedure or surgery
Transient elastography (Fibroscan®) is an affordable and noninvasive tool for measuring liver stiffness as a predictor of liver fibrosis. Since Fibroscan® measures liver's stiffness, its utility is not limited to fibrosis, and has been extended to other conditions that would increase the liver's stiffness, such as amyloidosis (Loustaud-Ratti et al. Amyloid 2011) and perhaps iron overload.
| Study group | Role | What they receive |
|---|---|---|
| Fibroscan | Other | Subjects enrolled will undergo Fibroscan. It is an affordable and noninvasive tool for measuring liver stiffness as a predictor of liver fibrosis. Fibroscan reading will be collected at the Gastroenterologist's (Dr. Ko) outpatient clinic (Pacific Gastroenterology Associates) where a qualified research nurse/assistant will perform the scan under supervision of the physician. Anticipated timing of this procedure will be October to December 2013 Receives: Procedure: Fibroscan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Fibroscan reading collected at the Gastroenterologist's outpatient clinic
Fibroscan results will be compared to that of T2* MRI, R2 MRI (FerriScan) and serum ferritin using linear regression models to determine if there is any correlation between FibroScan® results and liver iron concentration, which is indirectly measured with MRI and serum ferritin.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2014 (estimated)
- Study startOct 2013
- Primary completionDec 2015
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Hereditary AnemiasStudies of the same condition, open and under way.
- More trials run by University of British ColumbiaThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of British Columbia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Hatoon Ezzat, MD, Principal investigator, Department of Medicine, Division of Hematology St. Paul's Hospital, University of British ColumbiaHinhin Ko, MD, Principal investigator, Department of Medicine, Division of Gastroenterology, St. Paul's Hospital, University of British Columbia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02067130
- Sponsor's protocol ID
- H13-02149
- Status last verified
- Jan 2016
- First submitted
- 18 Feb 2014
- Last updated
- 14 Jan 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
Shander A, Sazama K. Clinical consequences of iron overload from chronic red blood cell transfusions, its diagnosis, and its management by chelation therapy. Transfusion. 2010 May;50(5):1144-55. doi: 10.1111/j.1537-2995.2009.02551.x. Epub 2010 Jan 15.
PubMed 20088842Jung KS, Kim SU. Clinical applications of transient elastography. Clin Mol Hepatol. 2012 Jun;18(2):163-73. doi: 10.3350/cmh.2012.18.2.163. Epub 2012 Jun 26.
PubMed 22893866Loustaud-Ratti VR, Cypierre A, Rousseau A, Yagoubi F, Abraham J, Fauchais AL, Carrier P, Lefebvre A, Bordessoule D, Vidal E, Sautereau D, Jaccard A. Non-invasive detection of hepatic amyloidosis: FibroScan, a new tool. Amyloid. 2011 Mar;18(1):19-24. doi: 10.3109/13506129.2010.543443. Epub 2011 Jan 10.
PubMed 21219116Remacha A, Sanz C, Contreras E, De Heredia CD, Grifols JR, Lozano M, Nunez GM, Salinas R, Corral M, Villegas A; Spanish Society of Blood Transfusion; Spanish Society of Haematology and Haemotherapy. Guidelines on haemovigilance of post-transfusional iron overload. Blood Transfus. 2013 Jan;11(1):128-39. doi: 10.2450/2012.0114-11. Epub 2012 Jul 4. No abstract available.
PubMed 22790272Gandon Y, Olivie D, Guyader D, Aube C, Oberti F, Sebille V, Deugnier Y. Non-invasive assessment of hepatic iron stores by MRI. Lancet. 2004 Jan 31;363(9406):357-62. doi: 10.1016/S0140-6736(04)15436-6.
PubMed 15070565Hou P, Popat UR, Lindsay RJ, Jackson EF, Choi H. A practical approach for a wide range of liver iron quantitation using a magnetic resonance imaging technique. Radiol Res Pract. 2012;2012:207391. doi: 10.1155/2012/207391. Epub 2012 Dec 11.
PubMed 23365743Argyropoulou MI, Astrakas L. MRI evaluation of tissue iron burden in patients with beta-thalassaemia major. Pediatr Radiol. 2007 Dec;37(12):1191-200; quiz 1308-9. doi: 10.1007/s00247-007-0567-1. Epub 2007 Aug 21.
PubMed 17710390
Study at a glance
From the registry record, last updated by the sponsor on 14 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Utility of Transient Elastography (Fibroscan) in Estimating Hepatic Iron Concentration in Comparison to MRI in Patients With Transfusion Dependent Hemoglobinopathies
- ClinicalTrials.gov ID
- NCT02067130
- Acronym / sponsor ID
- H13-02149
- Conditions
- Hereditary Anemias
- Keywords
- Beta Thalassemia Major
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of British ColumbiaUniversity, hospital or other
- Study start
- Oct 2013
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2016 (estimated)
- First posted
- 20 Feb 2014 (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.
In patients with hereditary anemias (e.g. thalassemias), defective red blood cells are produced due to an error in the genes, or DNA, that provide the instructions for their synthesis. As a result, hereditary anemias are characterized by chronically low hemoglobin, which is contained inside red blood cells and carries oxygen throughout the body. In more severe cases, patients are dependent on frequent blood transfusions to replenish the hemoglobin.
The body has limited ability to get rid of excess iron. However, with repeated blood transfusions, the iron level in the body builds up because the red blood cells contain iron as heme. Over time, the high level of iron accumulates in organs such as the heart, liver, and pancreas causing heart problems, liver failure, and diabetes. As a result, patients who receive multiple blood transfusions need to be monitored for iron overload, and be started on medical therapy in a timely fashion to prevent organ damage.
Liver is usually the first and the most affected organ by iron accumulation, so knowledge of its iron concentration provides estimate of total body iron load. Liver biopsy is the gold standard in measuring the iron concentration in the liver, but it is invasive and cannot be performed on routine basis. MRI is another option that can assess liver iron concentration non-invasively, and is currently recommended for monitoring iron load on a yearly basis. However, MRI has a high cost and is not easily accessible in Canada. The investigators aim to determine if transient elastography (Fibroscan), which is a form of ultrasound that measures liver stiffness, can accurately assess liver iron concentration.
Hypothesis:
Fibroscan reading correlates with MRI and serum ferritin in estimating hepatic iron concentration.
Detailed description
Inclusion criteria: All adult patients (age 19 or greater) with hereditary anemias requiring chronic blood transfusion at St. Paul's Hospital will be invited to participate in this study. The majority of patients will be β-Thalassemia Major. They will be given a pamphlet containing the details of the study and can contact the research assistant or clinic nurse for more information should they be interested.
Exclusion criteria: Patients with known Hepatitis B positive, known Hepatitis C positive, known HIV positive, known liver cirrhosis, known primary liver disease such as Wilson's disease and hereditary hemochromatosis are excluded from the study.
Written consent will be obtained from all participants by the clinic nurse/research assistant prior to enrollment.
Study procedures:
Data to be collected retrospectively from patient charts (St. Paul's Hospital's EMR/Sunrise Clinical Manager and paper chart) include: baseline demographic data (age, gender, hematological condition), medical comorbidities and complications related to iron overload (Diabetes, hypothyroidism, cardiomyopathy/arrhythmia and congestive heart failure, hypogonadotropic hypogonadism, osteopenia and osteoporosis syndrome..etc), current medications including use of iron chelators such as desferrioxamine, deferasirox, deferiprone or combination therapy, viral hepatitis status (B and C) and date of test, liver cirrhosis status (stage), liver biopsy result (iron concentration) and date of procedure. Patients with chronic transfusion requirement usually undergo annual MRI at the beginning of the year to estimate hepatic iron concentration as per standard practice. This year (2013), R2 MRI (FerriScan) will also be available for the first time to all patients in BC as part of routine monitoring for iron overload. Details/results from both techniques (i.e. same images collected in one scan, but analyzed differently using R2 and T2* algorithms) will be collected.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fibroscan | Other | Procedure: FibroscanSubjects enrolled will undergo Fibroscan. It is an affordable and noninvasive tool for measuring liver stiffness as a predictor of liver fibrosis. Fibroscan reading will be collected at the Gastroenterologist's (Dr. Ko) outpatient clinic (Pacific Gastroenterology Associates) where a qualified research nurse/assistant will perform the scan under supervision of the physician. Anticipated timing of this procedure will be October to December 2013 |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- All adult patients (age 19 or greater) with hereditary anemias requiring chronic blood transfusion at St. Paul's Hospital will be invited to participate in this study. The majority of patients will be β-Thalassemia Major.
Key exclusion criteria (5 listed)
- Known Hepatitis B positive
- Known Hepatitis C positive
- Known HIV positive
- Known liver cirrhosis
- Known primary liver disease such as Wilson's disease and hereditary hemochromatosis
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 |
|---|---|---|
| FibroscanTransient elastography (Fibroscan®) is an affordable and noninvasive tool for measuring liver stiffness as a predictor of liver fibrosis. Since Fibroscan® measures liver's stiffness, its utility is not limited to fibrosis, and has been extended to other conditions that would increase the liver's stiffness, such as amyloidosis (Loustaud-Ratti et al. Amyloid 2011) and perhaps iron overload. | Procedure or surgery | Fibroscan |
Primary outcome
Fibroscan reading collected at the Gastroenterologist's outpatient clinic
Time frame: 1 year
Fibroscan results will be compared to that of T2* MRI, R2 MRI (FerriScan) and serum ferritin using linear regression models to determine if there is any correlation between FibroScan® results and liver iron concentration, which is indirectly measured with MRI and serum ferritin.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2016.
- Background: PubMed 20088842Shander A, Sazama K. Clinical consequences of iron overload from chronic red blood cell transfusions, its diagnosis, and its management by chelation therapy. Transfusion. 2010 May;50(5):1144-55. doi: 10.1111/j.1537-2995.2009.02551.x. Epub 2010 Jan 15.
- Background: PubMed 22893866Jung KS, Kim SU. Clinical applications of transient elastography. Clin Mol Hepatol. 2012 Jun;18(2):163-73. doi: 10.3350/cmh.2012.18.2.163. Epub 2012 Jun 26.
- Background: PubMed 21219116Loustaud-Ratti VR, Cypierre A, Rousseau A, Yagoubi F, Abraham J, Fauchais AL, Carrier P, Lefebvre A, Bordessoule D, Vidal E, Sautereau D, Jaccard A. Non-invasive detection of hepatic amyloidosis: FibroScan, a new tool. Amyloid. 2011 Mar;18(1):19-24. doi: 10.3109/13506129.2010.543443. Epub 2011 Jan 10.
- Background: PubMed 22790272Remacha A, Sanz C, Contreras E, De Heredia CD, Grifols JR, Lozano M, Nunez GM, Salinas R, Corral M, Villegas A; Spanish Society of Blood Transfusion; Spanish Society of Haematology and Haemotherapy. Guidelines on haemovigilance of post-transfusional iron overload. Blood Transfus. 2013 Jan;11(1):128-39. doi: 10.2450/2012.0114-11. Epub 2012 Jul 4. No abstract available.
- Background: PubMed 15070565Gandon Y, Olivie D, Guyader D, Aube C, Oberti F, Sebille V, Deugnier Y. Non-invasive assessment of hepatic iron stores by MRI. Lancet. 2004 Jan 31;363(9406):357-62. doi: 10.1016/S0140-6736(04)15436-6.
- Background: PubMed 23365743Hou P, Popat UR, Lindsay RJ, Jackson EF, Choi H. A practical approach for a wide range of liver iron quantitation using a magnetic resonance imaging technique. Radiol Res Pract. 2012;2012:207391. doi: 10.1155/2012/207391. Epub 2012 Dec 11.
- Background: PubMed 17710390Argyropoulou MI, Astrakas L. MRI evaluation of tissue iron burden in patients with beta-thalassaemia major. Pediatr Radiol. 2007 Dec;37(12):1191-200; quiz 1308-9. doi: 10.1007/s00247-007-0567-1. Epub 2007 Aug 21.
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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.
Hatoon Ezzat, MD principal investigator
Department of Medicine, Division of Hematology St. Paul's Hospital, University of British Columbia
Hinhin Ko, MD principal investigator
Department of Medicine, Division of Gastroenterology, St. Paul's Hospital, University of British Columbia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fibroscan in people with Hereditary Anemias, run by University of British Columbia at 1 site in Canada.
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 1 inclusion and 5 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 14 Jan 2016. 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 Canada. 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?
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?
Fibroscan reading collected at the Gastroenterologist's outpatient clinic (time frame: 1 year). 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 NCT02067130, which also lists 7 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 14 Jan 2016.