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

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT00564941

Collaborator

(none)

309

Enrollment

Locations

Arm

Duration (Months)

77.3

Patients Per Site

1.8

Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndromes Beta-Thalassemia	Drug: deferasirox	Phase 4

Study Design

Study Type:

Interventional

Anticipated Enrollment :

309 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Dec 1, 2007

Actual Primary Completion Date :

Jul 1, 2011

Actual Study Completion Date :

Jul 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Deferasirox	Drug: deferasirox Other Names: ICL670

Arm

Intervention/Treatment

Experimental: Deferasirox

Drug: deferasirox

Other Names:

ICL670

Outcome Measures

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

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 [during the treatment (9 months)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	Country
1	Novartis Investigative Site	Budapest	Hungary
2	Novartis Investigative Site	Debrecen	Hungary
3	Novartis Investigative Site	Pecs	Hungary
4	Novartis Investigative Site	Szeged	Hungary