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

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00564941

Purpose

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

Condition	Intervention	Phase
Myelodysplastic Syndromes Beta-Thalassemia	Drug: deferasirox	Phase IV

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Anemia Blood Transfusion and Donation Thalassemia

Drug Information available for: Deferasirox

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-Thalassaemia Patients) With Chronic Iron Overload

Further study details as provided by Novartis:

Primary Outcome Measures:

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) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

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) ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	309
Study Start Date:	December 2007

Arms	Assigned Interventions
1: Experimental	Drug: deferasirox

Eligibility

Ages Eligible for Study:	18 Years to 80 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00564941

Contacts

Contact: Novartis

+41 61 324 1111

Locations

Hungary
Novartis Investigative Site	Recruiting
Budapest, Hungary
Novartis Investigative Site	Recruiting
Debrecen, Hungary
Novartis Investigative Site	Not yet recruiting
Pecs, Hungary
Novartis Investigative Site	Not yet recruiting
Szeged, Hungary

Sponsors and Collaborators

Novartis

Investigators

Study Chair:

Novartis

More Information

Responsible Party:	Novartis ( External Affairs )
Study ID Numbers:	CICL670AHU02
Study First Received:	November 28, 2007
Last Updated:	January 9, 2009
ClinicalTrials.gov Identifier:	NCT00564941
Health Authority:	Hungary: National Institute of Pharmacy

Keywords provided by Novartis:

Myelodysplastic Syndromes
beta-Thalassemia
Iron Overload
Iron Chelating Agents
deferasirox

Study placed in the following topic categories:

Myelodysplastic syndromes
Metabolic Diseases
Precancerous Conditions
Deferasirox
Hematologic Diseases
Beta-thalassemia
Myelodysplasia
Myelodysplastic Syndromes
Anemia
Anemia, Hemolytic
Iron Metabolism Disorders
Thalassemia

Anemia, Hemolytic, Congenital
Thalassemia minor
Preleukemia
Genetic Diseases, Inborn
Beta-Thalassemia
Hemoglobinopathies
Iron Overload
Metabolic disorder
Hemoglobinopathy
Bone Marrow Diseases
Iron

Additional relevant MeSH terms:

Neoplasms
Pathologic Processes
Disease
Molecular Mechanisms of Pharmacological Action

Syndrome
Iron Chelating Agents
Chelating Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009