cardiac iron overload MedDRA version: 9.1 Level: LLT Classification code 10019024 Term: Haemosiderosis
Conditions
Interventions
Trade Name: EXJADE
Pharmaceutical Form: Tablet
INN or Proposed INN: deferasirox
CAS Number: 201530-41-8
Current Sponsor code: ICL670A
Concentration unit: mg milligram(s)
Concentration type: equal
Con
Sponsors
NOVARTIS FARMA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Both sexes, aged ≥10 years β-thalassemic patients receiving regular transfusions with an history of at least 20 transfusional events Patients naive for deferasirox Serum ferritin levels ≥1000ng/mL (average of the last 6 months assays); Cardiac MRI T2* >5 and =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients that have already started deferasirox therapy Existing contraindications to the performance of MRI procedure, including presence of metallic prostheses not compatible with the MRI procedure other than those approved as safe for use in MRI scanners (e.g., some types of aneurysm clips, shrapnel in proximity to vital organs such as the retina), pacemaker, intractable claustrophobia and incapability to follow the instruction for the correct performance of the MRI (e.g. to maintain a supine position, to hold breath, etc.) Clinical conditions requiring intensive chelating therapy on the basis of Investigator?s judgment Severe liver impairment of ALT (SGOT) and/or AST (SGOT) > 5 times upper limit of the normal range (ULN) Uncontrolled systemic hypertension Estimated creatinine clearance <60 mL/min History of nephrotic syndrome History of clinically significant ocular toxicity related to the chelating therapy Psychiatric disorders or drug dependence that might impair the capability of the patient to provide their Informed Consent or to be administered the study treatment Pregnancy or breastfeeding Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or elimination of the any drugs, particularly: 1) History of intestinal inflammatory disease, gastritis, ulceration, gastro-intestinal and/or rectal bleeding 2) History of major gastro-intestinal surgery, such as gastrectomy, gastro-enterostomy, intestinal resection 3) History of pancreatic damage or pancreatitis; signs of pancreatic failure, such as alteration of lipases or amylases serum levels 4) History or presence of renal insufficiency as indicated by alteration of serum creatinine or BUN levels 5) History of urinary obstruction or voiding difficulty Patient judged potentially non-compliant and/or non-cooperative with the study procedures Women of childbearing potential not using the contraception method(s) specified in this study (specify), as well as women who are breastfeeding Known sensitivity to study drug(s) or class of study drug(s) Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study (specify as required) Use of any other investigational agent in the last 30 days.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the change in iron overload measured by cardiac MRI T2* after 52 weeks of treatment with deferasirox in &#946;-thalassemic patients affected by transfusion-dependent iron overload and T2* >5 msec and <20msec;Secondary Objective: To evaluate the change in iron overload measured by cardiac MRI T2* after 24 weeks of treatment with deferasirox. To evaluate the change of LVEF and of other cardiac function parameters by MRI (left ventricular end-systolic -LVESV- and end-diastolic volume- LVEDV, left ventricular mass) after 24 and 52 weeks of treatment with deferasirox. To evaluate the correlation of cardiac T2* with basal liver T2* and their subsequent modifications after 24 and 52 weeks of treatment. To evaluate the correlation between cardiac T2* and basal levels of serum ferritin (SF) and their subsequent modifications after 24 and 52 weeks of treatment. To evaluate NTBI species from baseline over 52 weeks of treatment with deferasirox.;Primary end point(s): Primary: Change T2* after 52 weeks of treatment according to baseline T2* value. | — |
Countries
Italy
Outcome results
None listed