Cardiac T2* in Beta-Thalassemia Patients on Deferasirox Treatment

Condition(s) targeted: Beta-Thalassemia; Iron Overload

Intervention: Deferasirox (Drug)

Phase: Phase 2

Status: Active, not recruiting

Sponsored by: Novartis

Official(s) and/or principal investigator(s):
Thomas Coates, MD, Principal Investigator, Affiliation: Childresn's Hospital of Los Angeles
Alexis Thompson, MD, Principal Investigator, Affiliation: Children's Memorial Hospital of Chicago
Paul Harmatz, MD, Principal Investigator, Affiliation: Children's Hospital and Research Center at Oakland

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

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

Study design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Primary outcome: Cardiac Iron in B thalassemia patients

Secondary outcome:

Safety and tolerabilty of deferasirox

Changes in liver iron concentration (LIC)

Changes in ventricular ejection fraction

Ferritin, NTBI (LPI and DCI)& iron metabolism parameters

Compliance with deferasirox

Minimum age: 10 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

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

Childrens Hospital of Los Angeles, Los Angeles, California 90027, United States

Children's Hospital and Research Center at Oakland, Oakland, California 94609, United States

Children's Memorial Hospital, Chicago, Illinois 60614, United States

Starting date: February 2006
Last updated: June 4, 2008