NCT01114776

Multi-Center Study of Iron Overload: Pilot Study

Pilot Study Examining Mechanisms of Iron Trafficking and Extra-hepatic Iron Distribution in Sickle Cell Disease, Thalassemia, and Other Iron Loading Anemias

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 20 (actual)
• Sponsor: UCSF Benioff Children's Hospital Oakland · Academic / Other
• Sex: All
• Age: 16 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to initiate pilot studies to demonstrate that a sufficient number of iron-overloaded thalassemia, SCD and DBA populations with similar duration of chronic transfusion, and age at start of transfusions would be available for a confirmatory study and to validate that proposed multicenter MRI and biochemical studies can be completed. The study will examine the hypothesis that a chronic inflammatory state in Sickle Cell Disease (SCD) leads to hepcidin- and cytokine-mediated iron withholding within the RES (reticuloendothelial system), lower plasma NTBI (non transferrin bound iron) levels, less distribution of iron to the heart in SCD.

Detailed description

A detailed iron burden, transfusion and chelation history will be obtained from chart review or from participant recall. Iron burden data will include: 1) documentation of liver iron, and 2) average annual ferritin values. Transfusion data will include: (1) age at onset of regular transfusions, (2) years of chronic transfusion therapy, and (3) pre-transfusion Hb calculated as average of all assessments for each year. MRI will be performed measuring pituitary, cardiac, and liver iron. Laboratory samples should be obtained pre-transfusion and mid-cycle. All interviews, exams, laboratory tests, study procedures and MRI assessments should be completed within a 0 to 12 weeks time span. In addition, a healthy control group will also be recruited with similar age, gender, and ethnicity as the disease groups.

Conditions

• Sickle Cell Disease
• Thalassemia
• Diamond-Blackfan Anemia

Timeline

• Start date: 2009-11-01
• Primary completion: 2013-07-31
• Completion: 2013-09-30
• First posted: 2010-05-03
• Last updated: 2020-09-24

Locations

3 sites across 3 countries: United States, Germany, United Kingdom