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

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

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Key information

Conditions

Myelodysplastic Syndromes Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Beta-Thalassemia Bone Marrow Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases Thalassemia

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Novartis Investigative Site, Budapest, Hungary

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Deferasirox

Drug

Other names: ICL670

Primary outcomes

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

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

Secondary outcomes

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

Time frame: during the treatment (9 months)

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Important dates

Study start: 2007
Primary completion: 2011
Study completion: 2011
First posted: Nov 29, 2007
Registry last updated: Feb 24, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed