Cardiac T2* in Beta-thalassemia Patient… | Clinical Trial

Key information

Conditions

Beta-Thalassemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases Thalassemia

Age range

10 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Childrens Hospital of Los Angeles, Los Angeles, California, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Male or female β-thalassemia outpatients on chronic transfusion therapy (defined as > 8 transfusions per year)
Lifetime minimum of 100 previous packed red blood cell transfusions
Patients currently on chelation therapy will require a one day wash out prior to the first dose of study drug
Age ≥ 10 years
Sexually active females of childbearing potential must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy.

Exclusion criteria

Ejection Fraction < 56 % measured using steady-state free precession imaging by MRI
Contraindication to MRI, including cardiac pacemaker, brain aneurysm clip, implanted neurostimulator, insulin pump, cochlear implant, metal slivers in the eyes, intrauterine device or any other MRI incompatible metal implants or intractable claustrophobia
Abnormal laboratory values as defined by the protocol
Clinical or laboratory evidence of active Hepatitis B or Hepatitis C
History of HIV positive test result (ELISA or Western blot)
Uncontrolled systemic hypertension
Second or third degree A-V block
Life-threatening arrhythmias, including sustained ventricular tachycardia and aborted sudden death, within the last year
History of cardiac conditions or unstable cardiac disease not controlled by standard medical therapy
History of clinically relevant ocular toxicity related to iron chelation
Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
Pregnancy or breast feeding (documented negative pregnancy test required for study entry)
Patients enrolled in an ongoing clinical trial of deferasirox (ICL670) cannot be withdrawn in order to participate in this study
Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug
History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
Other inclusion/exclusion criteria may apply

Treatment and study plan

Deferasirox

Drug

Oral deferasirox 30mg/kg/day once per day for 77 weeks.

Primary outcomes

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).

Secondary outcomes

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.

Sponsors and collaborators

Lead sponsor

Novartis

Industry

Registry information

Official study title

An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months

Important dates

Study start: 2006
Primary completion: 2009
Study completion: 2009
First posted: Mar 15, 2007
Registry last updated: Jun 14, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed