This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

Status

Completed

Phases

Phase 4

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01250951

Enrollment

111

Registered

2010-12-01

Start date

2009-12-31

Completion date

2011-09-30

Last updated

2016-12-12

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

Conditions

Myelodysplastic Syndrome, Thalassemia

Keywords

Iron Overload, hemotranfusion, deferasirox, ferritin, Rare anemia, Transfusional Iron Overload

Brief summary

This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.

Interventions

DRUGDeferasirox

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Age ≥ 2 years 2. Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia) 3. ECOG Performance Status ≤ 2 4. Transfusion overload confirmed with ferritin level \>1000 µg/l. 5. No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure). 6. Serum creatine level \> ULN 7. No proteinuria 8. Liver enzymes level \< 5 ULN. 9. No pregnancy or lactation 10. Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.

Exclusion criteria

1. Age \< 2 years 2. No iron overload (Ferritin level \<1000 µg/l). 3. Primary iron overload (hereditary hemochromatosis) 4. Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure) 5. Elevated serum creatinine \> ULN or/and proteinuria 6. Liver enzymes level \>5 ULN. 7. Pregnancy or lactation. Other protocol-defined inclusion/

Design outcomes

Primary

Measure	Time frame
changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade	Baseline assessment is followed by monthly assessments for up to 1 year

Secondary

Measure	Time frame
changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination	Baseline assessment is followed by monthly assessments for up to 1 year.
changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade	at baseline and 1 at year (at the end of study).
Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.	From the start of study up to 1 year

Countries

Russia

Outcome results

None listed

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