Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndrome and Transfusion-dependent Iron Overload

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT00481143

Collaborator

(none)

Enrollment

Location

Arm

Study Details

Study Description

Brief Summary

The purpose of this study is to investigate the effects of iron chelation using deferasirox in low and INT-1 risk (referring to the international prognostic scoring system, IPSS) MDS patients who show signs of iron overload due to repeated blood transfusions.

This trial is not recruiting patients in the United States.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndromes Transfusion Dependent Iron Overload	Drug: ICL670/Deferasirox	Phase 4

Study Design

Study Type:

Interventional

Actual Enrollment :

63 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload

Study Start Date :

May 1, 2007

Actual Primary Completion Date :

Nov 1, 2010

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: deferasirox

Drug: ICL670/Deferasirox

Other Names:

ICL670

Outcome Measures

Primary Outcome Measures

To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox [52 weeks]

Secondary Outcome Measures

Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs) [52 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

MDS patients presenting with low or intermediate-1 IPSS risk and transfusional iron overload
Patients of either gender and age ≥ 18 years
History of at least 20 units of red blood cell transfusions or 100mL/kg of prepacked red blood cells (PRBCs)
Patients can be either naïve to iron chelation or have had prior treatment with deferoxamine (DFO) or deferiprone (L1)
Females of childbearing potential must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Exclusion Criteria:

Non-transfusion related iron overload
Treatment with deferasirox (ICL670) before study start
Patients with a concomitant malignant disease
Patients with out of range lab values
History of nephrotic syndrome
Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drug within the past 7 days
Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug.
Patients with active uncontrolled infectious disease
Pregnancy or breast feeding