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.

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01250951

Collaborator

(none)

111

Enrollment

Locations

Arm

Duration (Months)

Patients Per Site

1.8

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndrome Thalassemia	Drug: Deferasirox	Phase 4

Study Design

Study Type:

Interventional

Actual Enrollment :

111 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Dec 1, 2009

Actual Primary Completion Date :

Jul 1, 2011

Actual Study Completion Date :

Sep 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Deferasirox	Drug: Deferasirox Other Names: ICL670

Arm

Intervention/Treatment

Experimental: Deferasirox

Drug: Deferasirox

Other Names:

ICL670

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	Country
1	Novartis Investigative Site	Moscow	Russian Federation
2	Novartis Investigative Site	Saint-Petersburg	Russian Federation
3	Novartis Investigative Site	St. Petersburg	Russian Federation