Whole liver iron overload measured by the biosusceptometer Magnetic Iron Detector (MID) in thalassemia and MDS patients treated with deferasirox - ND

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: Not specified

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

Non transfusional hemosiderosis
Renal impairment and/or with estimated creatinine clearance <60 ml/min at baseline
Serum creatinine above the upper limit of normal range at screening
Significant proteinuria as indicated by urinary protein/creatinine ratio ≥ 1 in a non-first void urine sample at baseline
History of nephrotic syndrome
Severe liver impairment and/or ALT (SGPT) and AST (SGOT) >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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 <br> Liver fibrosis assessment using FibroScan (EchoSens, Paris, France)<br> Assessment of serum ferritin value<br> Assessment of cardiac iron overload (MRI T2*) and cardiac function<br> Evaluation of electric conductivity of abdominal tissues to quantify and differentiate hepatic steatosis in order to assess the influence on the drug efficacy<br> 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.

Secondary Outcome Measures