Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
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|ClinicalTrials.gov Identifier: NCT00447694|
Recruitment Status : Completed
First Posted : March 15, 2007
Results First Posted : June 7, 2021
Last Update Posted : June 14, 2021
|Condition or disease||Intervention/treatment||Phase|
|Beta-thalassemia Iron Overload||Drug: Deferasirox||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months|
|Study Start Date :||February 2006|
|Actual Primary Completion Date :||November 2009|
|Actual Study Completion Date :||November 2009|
Experimental: deferasirox every day for 77 weeks
Participants received Deferasirox 30 milligrams per kilogram per day (mg/kg/day) orally once daily (OD), 30 minutes before breakfast, preferably around the same time every morning if possible. Deferasirox tablets were dropped into water or orange juice, or apple juice and stirred until completely dispersed. For doses less than 1 gram (g), tablets were dissolved in at least 100 milliliter (mL) of liquid; for doses of 1 to 3 g, tablets were dissolved in at least 200 mL. After tablets were fully disintegrated, the liquid was promptly consumed.
Oral deferasirox 30mg/kg/day once per day for 77 weeks.
- Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2* [ Time Frame: From Baseline to 25, 49, 77 Week ]Cardiac T2* was measured in the short axis plane at the widest point of a 4-chamber localizer using custom breath-hold R2* gradient echo sequences modeled after techniques used by Anderson et al (2001) and Westwood et al (2003).
- Change From Baseline in Liver Iron Concentration (LIC) Was Measured by MRI R2 From Absolute Change From Baseline to 101 Weeks [ Time Frame: From Baseline to 25, 49, 77 Week ]MRI evaluation of liver iron concentration has been validated by liver biopsy (St Pierre et al 2005). Studies comparing T2* values of liver iron concentration (LIC) with LIC as assessed by biopsy have confirmed that T2* values reflect liver iron content (Wood et al 2003b). Direct tissue-validation of cardiac T2* measurements in humans has not been performed because endomyocardial biopsy is a dangerous and unreliable indicator of cardiac iron overload (Olson et al 1989, Fitchett et al 1980). However, it has been shown that cardiac T2* accurately reflects cardiac iron in a gerbil iron cardiomyopathy model (Wood et al 2004b). T2* measurements have shown excellent inter-scanner and inter-exam reproducibility, making them suitable for longitudinal monitoring (Westwood et al 2003a, Westwood et al 2003b).
- Left Ventricular Ejection Fraction (LVEF) and Change in Left Ventricular Ejection Fraction From Baseline to 101 Weeks [ Time Frame: From Baseline to 25, 49, 77 Week ]Magnetic resonance imaging (MRI)-measured cardiac T2* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.
- Serum Ferritin and Changes From Baseline in Serum Ferritin During Study [ Time Frame: From Baseline to 25, 49, 77 Week ]Serum ferritin will be assessed at each study visit. Analysis was performed in Completer population consists of those participants who had a Week 77 MRI.
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): NCT00447694
|United States, California|
|Childrens Hospital of Los Angeles|
|Los Angeles, California, United States, 90027|
|Children's Hospital and Research Center at Oakland|
|Oakland, California, United States, 94609|
|United States, Illinois|
|Children's Memorial Hospital|
|Chicago, Illinois, United States, 60614|
|Principal Investigator:||Thomas Coates, MD||Childresn's Hospital of Los Angeles|
|Principal Investigator:||Alexis Thompson, MD||Children's Memorial Hospital of Chicago|
|Principal Investigator:||Paul Harmatz, MD||Children's Hospital and Research Center at Oakland|