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

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborators:
University College London (UCL) Cancer Institute
Universitätsklinikum Hamburg-Eppendorf
Medical University Innsbruck
Information provided by (Responsible Party):
Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier:
NCT01913548
First received: July 30, 2013
Last updated: NA
Last verified: July 2013
History: No changes posted
  Purpose

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.


Condition
Sickle Cell Disease
Thalassemia
Diamond-Blackfan Anemia

Study Type: Observational
Study Design: Observational Model: Case Control
Time Perspective: Prospective
Official Title: Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY

Resource links provided by NLM:


Further study details as provided by Children's Hospital & Research Center Oakland:

Primary Outcome Measures:
  • Identification of iron overloaded patients with Sickle Cell Disease and Thalassemia eligible for future study of iron deposition and biochemical mechanisms [ Time Frame: March 2010 - July 2013 ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 100
Study Start Date: March 2010
Estimated Study Completion Date: October 2013
Primary Completion Date: July 2013 (Final data collection date for primary outcome measure)
Groups/Cohorts
Sickle Cell Disease (SCD)
Patients with sickle cell diseases, 16 years or older with 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; and iron overload documented by either liver biopsy, MRI or SQUID with estimated LIC (liver iron content) of greater than 7 mg/g dry wt in the previous 6 months or ferritin level greater than 1500mg/dl.
Thalassemia Major (TM)
Patients with β-thalassemia major and transfusion-dependent E-beta THAL. 16 years or older with 10-20 years of chronic transfusion (defined above), 0 to 9 years old at the initiation of chronic transfusions, 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.
Diamond Blackfan Anemia (DBA)
Patients with DBA, 16 years or older with 10-20 years of transfusion, 0 to 9 years old at the initiation of chronic transfusions, 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.

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.

  Eligibility

Ages Eligible for Study:   16 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

There is no gender predisposition for sickle cell disease, thalassemia major, or Diamond-Blackfan anemia. Sickle cell anemia most frequently affects people of African descent, thalassemia affects people of Mediterranian, northern African, Southeast Asia and Indian descent. Diamond-Blackfan anemia occurs across all racial and ethnic groups.

Criteria

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/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, 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01913548

Locations
United States, California
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
United States, Georgia
Georgia Regents University
Augusta, Georgia, United States, 30912
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, North Carolina
Adult Comprehensive Sickle Cell Center, Duke University Medical Center
Durham, North Carolina, United States, 27710
United States, Pennsylvania
Thomas Jefferson SCD Program
Philadelphia, Pennsylvania, United States, 19107
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105-3678
Germany
Universitätsklinikum Hamburg-Eppendorf
Hamburg-Eppendorf, Germany
United Kingdom
UCL Cancer Institute
London, United Kingdom, WC1E 6BT
Sponsors and Collaborators
Children's Hospital & Research Center Oakland
University College London (UCL) Cancer Institute
Universitätsklinikum Hamburg-Eppendorf
Medical University Innsbruck
Investigators
Principal Investigator: Elliott Vichinsky, MD Children's Hospital & Research Center Oakland
Principal Investigator: John Porter, MD University College London Cancer Institute
  More Information

No publications provided

Responsible Party: Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier: NCT01913548     History of Changes
Other Study ID Numbers: 2010-019, 2R01DK057778-06A1
Study First Received: July 30, 2013
Last Updated: July 30, 2013
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Anemia
Anemia, Sickle Cell
Thalassemia
Anemia, Diamond-Blackfan
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Red-Cell Aplasia, Pure
Bone Marrow Diseases

ClinicalTrials.gov processed this record on August 28, 2014