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

Phase 4

Completed

• Conditions: Myelodysplastic Syndromes; Transfusion Dependent Iron Overload
• Interventions: Drug: ICL670/Deferasirox

• Registration Number: NCT00481143
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-05
• Study First Submitted: 2007-05-31
• Study First Submitted QC: 2007-05-31
• Study First Posted: 2007-06-01
• Primary Completion: 2010-11
• Status Verified: 2016-11
• Last Update Submitted: 2017-05-30
• Last Update Posted: 2017-05-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 63

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

Other protocol-defined inclusion/exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
deferasirox	ICL670/Deferasirox	-

Primary Outcome Measures

Name	Time	Method
To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox	52 weeks

Secondary Outcome Measures

Name	Time	Method
Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs)	52 weeks

Trial Locations

Locations (1)

Novartis Investigative Site; 🇩🇪 Berlin, Germany