Sickle Cell Disease Clinical Trial
— MCSIOOfficial title:
Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY
Verified date | September 2020 |
Source | UCSF Benioff Children's Hospital Oakland |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
The purpose of this study is to demonstrate that a sufficient number of iron-overloaded thalassemia (THAL), Sickle Cell Disease (SCD)and Diamond Blackfan Anemia (DBA) populations with similar duration of chronic transfusion, and age at start of transfusions would be available for a confirmatory study. The study will examine the hypothesis that a chronic inflammatory state in 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.
Status | Completed |
Enrollment | 423 |
Est. completion date | October 31, 2013 |
Est. primary completion date | August 31, 2013 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 16 Years and older |
Eligibility |
Inclusion Criteria: - 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. Exclusion Criteria: - Patients with HbSC, HbS/ß 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, THAL 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 |
Country | Name | City | State |
---|---|---|---|
Germany | Universitätsklinikum Hamburg-Eppendorf | Hamburg-Eppendorf | |
United Kingdom | UCL Cancer Institute | London | |
United States | Georgia Regents University | Augusta | Georgia |
United States | Children's Memorial Hospital | Chicago | Illinois |
United States | Adult Comprehensive Sickle Cell Center, Duke University Medical Center | Durham | North Carolina |
United States | St. Jude Children's Research Hospital | Memphis | Tennessee |
United States | Children's Hospital & Research Center Oakland | Oakland | California |
United States | Thomas Jefferson SCD Program | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
UCSF Benioff Children's Hospital Oakland | Medical University Innsbruck, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), Universitätsklinikum Hamburg-Eppendorf, University College London (UCL) Cancer Institute |
United States, Germany, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Identification of iron overloaded patients with Sickle Cell Disease and Thalassemia eligible for future study of iron deposition and biochemical mechanisms | Patients with similar duration of chronic transfusion and age at onset of chronic transfusion therapy will be identified from 10 participating centers. Detailed information on iron burden and transfusion, medical, and chelation histories will be obtained in order to establish a cohort of patients that could be available for a future powered study of extra-hepatic iron deposition and underlying biochemical mechanisms. | March 2010 - July 2013 |
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