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Iron Mediated Vascular Disease in Sickle Cell Anemia Patients

The recruitment status of this study is unknown because the information has not been verified recently.
Verified November 2011 by Children's Hospital Los Angeles.
Recruitment status was  Recruiting
Sponsor:
Information provided by (Responsible Party):
John C. Wood, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier:
NCT01239901
First received: November 12, 2010
Last updated: November 28, 2011
Last verified: November 2011
  Purpose

The purpose of this research study is to determine the frequency and severity of iron overload in patients with Sickle Cell Anemia and its relationship to blood vessel function. The investigators hypothesize that intermittent transfusions that these patients receive during hospitalizations produces significant iron overload and impairs blood vessel relaxation.


Condition
Sickle Cell Disease

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Cross-Sectional
Official Title: Iron-mediated Vascular Disease in Sickle Cell Disease.

Resource links provided by NLM:


Further study details as provided by Children's Hospital Los Angeles:

Primary Outcome Measures:
  • Liver iron concentration (LIC), pancreas R2*, and kidney R2*, measured by MRI [ Time Frame: 15 min MRI done completed during one time study visit ] [ Designated as safety issue: No ]
    Liver iron concentration (LIC) will be used as a surrogate for total body iron, pancreatic iron represents a surrogate for extravascular iron deposition, and renal iron as a surrogate for chronic intravascular hemolysis. In addition, labile plasma iron will be measured in blood plasma.


Secondary Outcome Measures:
  • Vascular function. [ Time Frame: Scheduled during one time study visit ] [ Designated as safety issue: No ]
    Measurements will be done through several procedures: Ultrasound of artery in upper arm, Ultrasound of artery in neck, and blood tests of ascorbate and hydrobiopterins.


Biospecimen Retention:   Samples With DNA

Red Blood Cells (RBCs), Peripheral Blood Mononuclear Cells (PBMCs), Plasma, Serum, and Urine


Estimated Enrollment: 150
Study Start Date: December 2009
Estimated Study Completion Date: October 2013
Estimated Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Detailed Description:

Patients with sickle cell anemia often require blood transfusion as part of the treatment for their disease. Each teaspoon of transfused blood contains about 5 mg of iron and the levels of iron in sickle cell patients increase rapidly with each transfusion. While iron is necessary for many bodily functions, too much iron damages blood vessels, liver, hormone producing glands (pancreas, pituitary and thyroid) and the heart. It is important to know how iron damages blood vessels because most of the problems experienced by sickle cell anemia patients (stroke, kidney failure, pulmonary hypertension, heart disease) result from blood vessel damage. In this trial, iron in the liver, pancreas, and kidney will be measured noninvasively by MRI while vascular function will be measured by ultrasound and tissue Doppler. Patients will be recruited primarily from the greater Los Angeles area, although patients from greater distances will be allowed to participate.

  Eligibility

Ages Eligible for Study:   13 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Our large and well-established sickle cell Center has strong ties to the SCD community in a large metropolitan area (Los Angeles), placing us in an ideal position to rapidly accrue sufficient subjects to study the prevalence of iron overload and determine the relation between directly measured iron loading, vascular responsiveness and clinically relevant biomarkers of vascular function.

Criteria

Inclusion Criteria:

  • Age > 13 years
  • Documented diagnosis of sickle cell anemia (SS, SC, Sß0, Sß+)
  • Transfused no more than 8 times in a year.

Exclusion Criteria:

  • Cardiac pacemaker, implantable neurostimulator or other MRI incompatible device.
  • History of extreme claustrophobia in MRI machine or other reason for inability to do MRI without sedation.
  • Inability to be positioned on the MRI table for sufficient time to complete the MRI exams.
  • Any medical or psychological condition that, in the opinion of the local investigator, would make it unsafe or ill-advised for the subject to participate.
  • Currently not receiving chronic transfusion therapy, defined as greater than 8 transfusions per year.
  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: NCT01239901

Contacts
Contact: Tatiana Hernandez, BA 323-361-8827 tahernandez@chla.usc.edu

Locations
United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Tatiana Hernandez, BA    323-361-8827    tahernandez@chla.usc.edu   
Principal Investigator: John C Wood, MD, PhD         
Sub-Investigator: Thomas D Coates, MD         
Sponsors and Collaborators
Children's Hospital Los Angeles
Investigators
Principal Investigator: John C Wood, MD, PhD Children's Hospital Los Angeles
  More Information

Publications:

Responsible Party: John C. Wood, Associate Professor of Pediatrics - Division of Cardiology, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT01239901     History of Changes
Other Study ID Numbers: 1 RC1 HL099412-01
Study First Received: November 12, 2010
Last Updated: November 28, 2011
Health Authority: United States: National Institutes of Health

Keywords provided by Children's Hospital Los Angeles:
sickle cell disease
sickle cell anemia
sickled cells
blood vessel
iron overload

Additional relevant MeSH terms:
Anemia, Sickle Cell
Vascular Diseases
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Cardiovascular Diseases
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on November 24, 2014