In- or outpatients with myelodysplasia or beta thalassaemia major, who have chronic iron overload, as a consequence of frequent blood transfusion
Conditions
Interventions
Trade Name: Exjade
Product Name: Exjade
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: deferasirox
CAS Number: 201530418
Current Sponsor code: ICL670
Other descriptive name: DEFERASIROX
Sponsors
Novartis Hungária Kft.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion •Serum ferritin> 1800 µg/L • Age: 18-80 years •Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells •Deferoxamin (Desferal®) therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason •ECOG performance status score between 0-2 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: •beta thalassaemia minor, •haemosiderosis caused by other than chronic transfusional iron overload, •patients with impaired renal function (Creatinin clearance 0.5 (mg/mg) in second-voiding urine samples taken at either visits 1 or 2. •History of non-compliance to medical regimens •Patients with positive test to HIV • Life expectancy of < 1year
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary objective: to evaluate the clinical response to treatment with Exjade® from the baseline to weeks 36 by following serum-ferritin change in patients with transfusion dependent chronic anaemias ( Myelodysplastic Syndrome, Beta-thalassaemia major patients) with chronic iron overload. ;Secondary Objective: Secondary objectives: to assess drug usage compliance, evaluate patient’s compliance by monthly counting returned study medication and retrospective interviews with patient/parent/caregiver, and assess safety data during 36 week long treatment with Exjade® in patients with transfusion dependent chronic anaemias ( Myelodysplastic Syndrome, Beta-thalassaemia major patients) with chronic iron overload. ;Primary end point(s): Assessment of the efficacy of the Exjade treatment in patient with transfusional hemosiderosis in MDS patients and beta thalassaemia major patients based on changes in serum ferritin level during the study from the baseline up to the 36. weeks of the treatment | — |
Countries
Hungary
Outcome results
None listed