Trial record 15 of 15 for:    Open Studies | "Hemochromatosis"

Liver Fibrosis in Sickle Cell Disease

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2014 by University of Miami
Sponsor:
Information provided by (Responsible Party):
Ofelia Alvarez, University of Miami
ClinicalTrials.gov Identifier:
NCT02007746
First received: December 6, 2013
Last updated: September 3, 2014
Last verified: September 2014
  Purpose

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.The primary hypothesis is that the results of FibroScan will correlate with the results of Ferriscan.


Condition Intervention
Sickle Cell Disease
Other: Liver transient elastography (Fibroscan)
Other: Ferriscan
Procedure: Liver biopsy

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Cross-Sectional
Official Title: Assessment of Liver Fibrosis in Patients With Sickle Cell Disease

Resource links provided by NLM:


Further study details as provided by University of Miami:

Primary Outcome Measures:
  • Liver transient elastography (FibroScan) of liver iron content and stiffness [ Time Frame: at imaging visit (3 minutes) ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • magnetic resonance imaging (MRI) measure of liver iron content and stiffness [ Time Frame: at imaging visit (about 30-60 minutes) ] [ Designated as safety issue: No ]
  • 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) ] [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: July 2012
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: July 2017 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Subjects with sickle cell disease
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.
Other: Liver transient elastography (Fibroscan)
Fibroscan will be performed as a measure of liver stiffness. The study will be obtained free of charge in this study.
Other Name: FibroScan
Other: Ferriscan
Ferriscan will be done to assess the quantity of liver iron.
Procedure: Liver biopsy
Liver biopsy will be done and the results will be compared to Fibroscan results.

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 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.

  Eligibility

Ages Eligible for Study:   10 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

30 subjects with sickle cell disease

Criteria

Inclusion Criteria:

  • pediatric patients age 10 years and older with sickle cell disease
  • meeting other criteria:

    1. history of chronic transfusion and iron overload and/or
    2. known liver disease related to sickle cell or iron overload
  • signed consent and assent (as applicable)

Exclusion Criteria:

  • children younger than 10 years
  • Pregnant females
  • Prisoners
  • Other causes of liver disease, unrelated to sickle cell or iron overload
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02007746

Contacts
Contact: Ofelia Alvarez, MD 305.243.0846 oalvarez2@med.miami.edu

Locations
United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Ofelia Alvarez, MD    305-243-0846    oalvarez2@med.miami.edu   
Sub-Investigator: Gaurav Saigal, MD         
Sub-Investigator: Eugene Schiff, MD         
Sponsors and Collaborators
University of Miami
Investigators
Principal Investigator: Ofelia Alvarez, MD University of Miami - Director Sickle Cell Services Pediatric Hematology/Oncology
  More Information

Publications:
Responsible Party: Ofelia Alvarez, Director, Sickle Cell Disease Center, Pediatric Hematology/Oncology, University of Miami
ClinicalTrials.gov Identifier: NCT02007746     History of Changes
Other Study ID Numbers: 20120222
Study First Received: December 6, 2013
Last Updated: September 3, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Miami:
sickle cell disease
hemochromatosis
liver fibrosis
liver cirrhosis
FibroScan
Ferriscan
liver iron

Additional relevant MeSH terms:
Anemia, Sickle Cell
Fibrosis
Liver Cirrhosis
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Pathologic Processes
Liver Diseases
Digestive System Diseases
Liver Extracts
Hematinics
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 11, 2014