Patients with diagnosis of thalassemia major with iron overload or regularly transfused MDS patients (Low/Intermediate-1 IPSS risk and 1 year life expectancy) MedDRA version: 9.1 Level: LLT Classification code 10010329 Term: Congenital anaemia
Conditions
Interventions
Trade Name: EXJADE
Pharmaceutical Form: Tablet
INN or Proposed INN: Deferasirox
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Sponsors
AZIENDA OSPEDALIERA OSPEDALI GALLIERA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Diagnosis of thalassemia major with iron overload or regularly transfused MDS patients (Low/Intermediate-1 IPSS risk and 1 year life expectancy) M/F adult patients (age ≥18 years) Patients starting/about to start or potentially eligible to the treatment with deferasirox according to locally approved label Female patients who are sexually active must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation Written informed consent by the patient in accordance with the national legislation Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Non transfusional hemosiderosis Renal impairment and/or with estimated creatinine clearance 5 ULN Patients with uncontrolled systemic hypertension Pregnant or breast feeding patients Hypersensitivity to the active substance or to any of the excipients Concomitant use of other iron chelating agents History of clinically relevant ocular and auditory toxicity related to iron chelation therapy Heart function: Left Ventricular Ejection Fraction (LEF) <50% assessed by echo or <60% by MRI and/or T2* <10 ms measured in the last year Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment Patients unable to undergo study assessments including MRI and MID e.g. patients who are claustrophobic to MRI or have metallic prostheses not compatible with both MRI and MID procedures or patients who are obese (exceeding the equipment limits of MRI) Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days. Patients being treated with experimental drugs for MDS, including thalidomide or analogues, azacitidine in the previous four weeks. These patients are eligible after a minimum washout period of four weeks Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as the following: - history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding - history of major gastrointestinal tract surgery such as gastrectomy, gastro-enterostomy or bowel resection - history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase - history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN - history of urinary obstruction or difficulty in voiding History of drug or alcohol abuse Patients with positive test to HIV History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative Patient unwilling to comply with the protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the iron chelation therapy efficacy with deferasirox by total liver iron overload assessment using MID.;Secondary Objective: Assessment of iron burden in abdomen (extra-hepatic site) through MRI Liver fibrosis assessment using FibroScan (EchoSens, Paris, France) Assessment of serum ferritin value Assessment of cardiac iron overload (MRI T2*) and cardiac function Evaluation of electric conductivity of abdominal tissues to quantify and differentiate hepatic steatosis in order to assess the influence on the drug efficacy Assessment of safety and tolerability of deferasirox treatment;Primary end point(s): To determine the iron chelation therapy efficacy with deferasirox by total liver iron overload assessment using MID. | — |
Countries
Italy
Outcome results
None listed