Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload
Information source: Novartis
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Transfusional Iron Overload; Transfusional Hemosiderosis
Intervention: Core Study: Deferasirox (Drug); Core Study: Deferoxamine (Drug); Extension: deferoxamine to deferasirox (Drug); Extension: deferasirox to deferoxamine (Drug); Deferasirox (Drug); Deferoxamine (Drug)
Phase: Phase 2
Status: Completed
Sponsored by: Novartis Pharmaceuticals Official(s) and/or principal investigator(s): Novartis Pharmaceuticals, Study Director, Affiliation: Novartis Pharmaceuticals
Summary
This is a clinical research study in patients who have iron overload in the heart due to
chronic blood transfusions.
The study will have 2 treatment groups and will compare the safety and efficacy of chelation
therapy with a medicine called deferasirox (ICL670) with another medicine called
deferoxamine (DFO). The study is aimed at finding out which of the two medicines is the best
for treating iron overload in the heart.
Patients will be treated for 12 months (core study phase). Patients who complete the core
study phase will be offered to continue their study treatment in a 12 months extension
phase. During the core and extension, the effects of treatment on iron overload in the heart
and the liver will be evaluated using specific magnetic resonance imaging (MRI) assessments.
Clinical Details
Official title: A Multicenter, Randomized, Open-label Phase II Trial Evaluating Deferasirox Compared With Deferoxamine in Patients With Cardiac Iron Overload Due to Chronic Blood Transfusions
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Core Study: Change From Baseline in Myocardial T2* (Magnetic Resonance T2-star (T2*) Technique for the Measurement of Tissue Iron) After 12 Months Treatment
Secondary outcome: Core Study: Cardiac Function After 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)Core Study: Cardiac Function After 6 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF) Core Study: Change From Baseline in Myocardial T2* After 6 Months Treatment Core Study: Cardiac Function After 6 and 12 Months Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Systolic Volume Indices (LVESVI) Core Study: Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI) Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Mass Indices (LVMI) Core Study: Cardiac Function and the Proportion of Patients Dropping Out Due to Cardiac Dysfunction After Treatment With Deferasirox vs. Deferoxamine Core Study: Safety and Tolerability of Deferasirox vs Deferoxamine Over the 12 Months Treatment Period. Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Area Under the Plasma Concentration-time Curve for a Dosing Interval (AUCtau) Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Cmax) Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Time Points of Concentration Data Extension Study: Change From Baseline in Myocardial T2* After 24 Months Treatment Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Ejection Fraction (LVEF) Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Systolic Volume Indices (LVESVI) Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI) Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Mass Indices (LVMI) Extension Study: The Cardiac Iron Concentration From T2* Values Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24 Extension Study: Change in Serum Ferritin From Baseline by Month Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Tmax)
Eligibility
Minimum age: 10 Years.
Maximum age: 65 Years.
Gender(s): Both.
Criteria:
Inclusion criteria:
- Male or female patients, aged 10 years and above, with β-thalassemia major or DBA or
sideroblastic anemia on chronic transfusion therapy, having given written consent to
participate in the study.
- Patients with cardiac iron as measured by a myocardial T2* value that is ≥ 6ms but
not ≥ 20 ms.
- Patients with a lifetime history of at least 50 units of red cell transfusions, and
must be receiving at least ≥10 units/yr of red blood cells transfusions.
- Patients with a left ventricular ejection fraction (LVEF) ≥ 56 % as determined by
cardiovascular magnetic resonance (CMR).
- Patients with liver iron content (LIC) value ≥ 3 mg Fe / g dw, as determined by liver
MRI.
Exclusion criteria:
- Patients with clinical symptoms of cardiac dysfunction.
- Patients unable to undergo study assessments including MRI
- Patients participating in another clinical trial or receiving an investigational
drug.
Other protocol-defined inclusion/exclusion criteria may apply.
Locations and Contacts
Novartis Investigative Site, Limassol 3304, Cyprus
Novartis Investigative Site, Cairo, Egypt
Novartis Investigative Site, Mansoura, Egypt
Novartis Investigative Site, Hazmiyeh, Lebanon
Novartis Investigative Site, Taipei 10002, Taiwan
Novartis Investigative Site, Bangkok 10330, Thailand
Novartis Investigative Site, Bangkok 10700, Thailand
Novartis Investigative Site, Adana 01330, Turkey
Novartis Investigative Site, Ankara 06100, Turkey
Novartis Investigative Site, Antalya 07070, Turkey
Novartis Investigative Site, Istanbul 34093, Turkey
Novartis Investigative Site, Izmir 35040, Turkey
Novartis Investigative Site, Dubai 9115, United Arab Emirates
Novartis Investigative Site, Leeds LS9 7TF, United Kingdom
Novartis Investigative Site, London N19 5NF, United Kingdom
Novartis Investigative Site, London NW1 2PJ, United Kingdom
Novartis Investigative Site, Cagliari, CA 09121, Italy
Novartis Investigative Site, Genova, GE 16128, Italy
Novartis Investigative Site, Nanning, Guangxi 530021, China
Novartis Investigative Site, Toronto, Ontario M5G 1X8, Canada
Novartis Investigative Site, Leeds, West Yorkshire LS9 7TF, United Kingdom
Additional Information
Starting date: November 2007
Last updated: August 13, 2014
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