Multi-Center Study of Iron Overload: Survey Study (MCSIO)

Sickle Cell Disease Clinical Trial

— MCSIO  

Official title:

Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01913548
• Other study ID #: 2010-019
• Secondary ID: 2R01DK057778-06A
• Status: Completed
• 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
• 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

Related Conditions & MeSH terms

• Anemia, Diamond-Blackfan
• Anemia, Sickle Cell
• Diamond-Blackfan Anemia
• Sickle Cell Disease
• Thalassemia

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