MedDRA version: 19.0 Level: LLT Classification code 10054658 Term: Thalassemia System Organ Class: 100000004850
Conditions
Sponsors
Novartis
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •- Male or female ß-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. Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 23 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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test required for study entry) •Patients enrolled in an ongoing clinical trial of deferasirox (ICL670) cannot be withdrawn in order to participate in this study •Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Cardiac Iron in B thalassemia patients ( Time Frame: From baseline to week 25 and weeks 49 and 77 of the trial );Secondary Objective: •Safety and tolerabilty of deferasirox [ Time Frame: throughout the study ] •Changes in liver iron concentration (LIC) [ Time Frame: From baseline to week 25 and weeks 49 and 77 of trial ] •Changes in ventricular ejection fraction [ Time Frame: From baseline to weeks 25, 49 and 77 ] •Ferritin, NTBI (LPI and DCI)& iron metabolism parameters [ Time Frame: From Baseline through week 25, and weeks 49 and 77 of trial ] •Compliance with deferasirox [ Time Frame: Throughout the study ] ;Primary end point(s): Cardiac Iron in B thalassemia patients;Timepoint(s) of evaluation of this end point: From baseline to week 25 and weeks 49 and 77 of the trial | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •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;Timepoint(s) of evaluation of this end point: •Safety and tolerabilty of deferasirox: throughout the study •Changes in liver iron concentration : From baseline to week 25 and weeks 49 and 77 of trial •Changes in ventricular ejection fraction: From baseline to weeks 25, 49 and 77 •Ferritin, NTBI (LPI and DCI)& iron metabolism parameters : From Baseline through week 25, and weeks 49 and 77 of trial •Compliance with deferasirox: Throughout the study | — |
Countries
United States
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed