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Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

Phase 4
18 Years
80 Years
Not Enrolling
Myelodysplastic Syndromes, Beta-Thalassemia

Thank you

Trial Information

Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

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

- 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/exclusion criteria may apply

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload

Outcome Time Frame:

monthly during the therapy and at the end of the treatment (aftr 9 months therapy)

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


Hungary: National Institute of Pharmacy

Study ID:




Start Date:

December 2007

Completion Date:

Related Keywords:

  • Myelodysplastic Syndromes
  • Beta-Thalassemia
  • Myelodysplastic Syndromes
  • beta-Thalassemia
  • Iron Overload
  • Iron Chelating Agents
  • deferasirox
  • Beta-Thalassemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Thalassemia
  • Iron Overload