Beta-Thalassemia, Myelodysplastic Syndromes
Conditions
Keywords
Myelodysplastic Syndromes, beta-Thalassemia, Iron Overload, Iron Chelating Agents, deferasirox
Brief summary
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
Interventions
DRUGdeferasirox
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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 * men and women * Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells * Deferoxamin therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason * Eastern Cooperative Oncology Group (ECOG) performance status score between 0-2 * written informed consent
Exclusion criteria
* beta thalassaemia minor, * haemosiderosis caused by other than chronic transfusional iron overload, * patients with impaired renal function (Creatinin clearance\< 60 ml/ min), * pregnancy, * lactation, * patient of childbearing potential unwilling to use contraceptive precautions * known hypersensitivity to deferasirox or any ingredients, * impaired hepatic function (SGOT,SGPT 5x above UNL). * Patients severely ill due to underlying disease progression or other severe concomitant disease. * Patients with poor prognosis of karyotype * patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition * History of nephrotic syndrome * Significant proteinuria * Patients with a previous history of clinically relevant ocular toxicity related to iron chelation * Patients with positive test to HIV Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload | monthly during the therapy and at the end of the treatment (aftr 9 months therapy) |
Secondary
| Measure | Time frame |
|---|---|
| patient's compliance during the study assessed by the number of the unused tablets returned by the patient safety assessed by patient laboratory data, adverse events, serious adverse events | during the treatment (9 months) |
Countries
Hungary
Outcome results
None listed