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

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

Status

Completed

Phases

Phase 4

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00481143

Enrollment

Registered

2007-06-01

Start date

2007-05-31

Completion date

Unknown

Last updated

2017-05-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Myelodysplastic Syndromes, Transfusion Dependent Iron Overload

Keywords

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)

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.

Interventions

DRUGICL670/Deferasirox

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

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 Other protocol-defined inclusion/

Design outcomes

Primary

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

Secondary

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

Countries

Germany

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026