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.

Novartis

Status and phase

Completed

Phase 4

Conditions

Thalassemia

Myelodysplastic Syndrome

Treatments

Drug: Deferasirox

Study type

Interventional

Funder types

Industry

Identifiers

NCT01250951

CICL670ARU01

Details and patient eligibility

About

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

Enrollment

111 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Age ≥ 2 years
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)
ECOG Performance Status ≤ 2
Transfusion overload confirmed with ferritin level >1000 µg/l.
No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure).
Serum creatine level > ULN
No proteinuria
Liver enzymes level < 5 ULN.
No pregnancy or lactation
Signed informed consent by adults. In case inclusion of children under 18 years old, the informed consent should be signed by parents.

Exclusion criteria

Age < 2 years
No iron overload (Ferritin level <1000 µg/l).
Primary iron overload (hereditary hemochromatosis)
Severe concomitant disease (uncontrolled diabetes mellitus, heart failure, renal failure)
Elevated serum creatinine > ULN or/and proteinuria
Liver enzymes level >5 ULN.
Pregnancy or lactation.

Other protocol-defined inclusion/exclusion criteria may apply