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

Novartis

Status and phase: Completed; Phase 4

Conditions: Beta-Thalassemia; Myelodysplastic Syndromes

Treatments: Drug: deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00564941

CICL670AHU02

Details and patient eligibility

About

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

Enrollment: 309 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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309 participants in 1 patient group

Deferasirox

Experimental group

Treatment:

Drug: deferasirox