Multi-Center Study of Iron Overload: Survey Study (MCSIO) (MCSIO)
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|ClinicalTrials.gov Identifier: NCT01913548|
Recruitment Status : Completed
First Posted : August 1, 2013
Last Update Posted : September 23, 2020
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|Condition or disease|
|Sickle Cell Disease Thalassemia Diamond-Blackfan Anemia|
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.
|Study Type :||Observational|
|Actual Enrollment :||423 participants|
|Official Title:||Modulation of Iron Deposition in Sickle Cell Disease and Other Hemoglobinopathies SURVEY STUDY|
|Study Start Date :||March 31, 2010|
|Actual Primary Completion Date :||August 31, 2013|
|Actual Study Completion Date :||October 31, 2013|
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.
- 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 ]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.
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|Ages Eligible for Study:||16 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01913548
|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|
|UCL Cancer Institute|
|London, United Kingdom, WC1E 6BT|
|Principal Investigator:||Elliott Vichinsky, MD||UCSF Benioff Children's Hospital Oakland|
|Principal Investigator:||John Porter, MD||University College London Cancer Institute|
|Responsible Party:||UCSF Benioff Children's Hospital Oakland|
|Other Study ID Numbers:||
2R01DK057778-06A1 ( U.S. NIH Grant/Contract )
|First Posted:||August 1, 2013 Key Record Dates|
|Last Update Posted:||September 23, 2020|
|Last Verified:||September 2020|
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Anemia, Hypoplastic, Congenital
Red-Cell Aplasia, Pure
Congenital Bone Marrow Failure Syndromes
Bone Marrow Failure Disorders
Bone Marrow Diseases