Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

Novartis

Status and phase: Completed; Phase 2

Conditions: Iron Overload; Beta-thalassemia

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00447694

CICL670AUS04

Details and patient eligibility

About

The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.

Enrollment: 30 patients

Sex: All

Ages: 10+ years old

Volunteers: No Healthy Volunteers

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• Patients enrolled in an ongoing clinical trial of deferasirox (ICL670) cannot be withdrawn in order to participate in this study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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

deferasirox every day for 77 weeks

Experimental group

Description:

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.

Treatment:

Drug: Deferasirox