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

Study Overview
=================
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.

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

Conditions
-----------------
Myelodysplastic Syndromes, Transfusion Dependent Iron Overload

Intervention / Treatment
-----------------
* Drug: ICL670/Deferasirox


Participation Criteria
=================
Eligibility Criteria
-----------------
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.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: deferasirox<br> | Drug: ICL670/Deferasirox<br> <br> * Other names: ICL670;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To assess iron chelation by comparing serum ferritin values at baseline vs. 52 weeks of treatment with deferasirox |  | 52 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of deferasirox assessed by monitoring and recording all adverse events (AEs) and serious adverse events (SAEs) |  | 52 weeks | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Myelodysplastic Syndrome, MDS, iron overload, chelation, chelators, ICL670, Deferasirox, serum ferritin, LIC, transfusional hemosiderosis, Refractory Anemia, RA, Refractory Anemia with Ringed Sideroblasts, RARS, blood transfusions, Mydelodysplastic Syndrome(s)