Multi-Center Study of Iron Overload: Pilot Study

Completed

Conditions studied: Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia

In brief

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.

Key facts

Study ID

NCT01114776

Run by

UCSF Benioff Children's Hospital Oakland

People needed

20

Starts

2009-11-01

Expected to finish

2013-09-30

Last updated by the study team

2020-09-24

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• 10-20 years of transfusion (defined as 0.2-0.6mg Fe/kg/day exposure with annual ferritin levels greater than 2500 in at least 60% of years of chronic transfusion);
• 0 to 9 years old at the initiation of chronic transfusions; no exchange transfusions in the previous 6 months
• iron overload documented by either liver biopsy, MRI or SQUID with estimated LIC of greater than 7 mg/g dry wt in the previous 6 months or ferritin level greater than 1500mg/dl.

You may not qualify if…

• Patients with HbSC, HbS/β thalassemia
• Pacemaker (active or inactive) or other implanted magnetic devices, severe claustrophobia, or other contraindications to MRI; Unable to remove ferro-magnetic objects from the body in regions to be imaged (e.g., jewelry or piercing)
• Presence of any other condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment;
• Any chronic inflammatory illness other than the SCD, TM or DBA;
• Any acute illness within a 14 day period prior to blood sampling;
• Patients receiving intensive chelation in the 6 months prior to enrollment including deferoxamine 24 hours per day, 7 days per week or combination treatment with 2 chelators
• Pregnancy

Where it is running

• Children's Hospital & Research Center Oakland — Oakland, California, United States
• Universitätsklinikum Hamburg-Eppendorf — Hamburg-Eppendorf, Germany
• UCL Cancer Institute — London, United Kingdom

Full record on ClinicalTrials.gov

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