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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01913548
Other study ID # 2010-019
Secondary ID 2R01DK057778-06A
Status Completed
Phase
First received
Last updated
Start date March 31, 2010
Est. completion date October 31, 2013

Study information

Verified date September 2020
Source UCSF Benioff Children's Hospital Oakland
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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.


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.


Recruitment information / eligibility

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

Study Design


Locations

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

Sponsors (5)

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

Countries where clinical trial is conducted

United States,  Germany,  United Kingdom, 

Outcome

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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