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.

Phase 4

Completed

• Conditions: Myelodysplastic Syndrome; Thalassemia
• Interventions: Drug: Deferasirox

• Registration Number: NCT01250951
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-12
• Study First Submitted: 2010-11-11
• Study First Submitted QC: 2010-11-29
• Study First Posted: 2010-12-01
• Primary Completion: 2011-07
• Study Completion: 2011-09
• Status Verified: 2011-12
• Last Update Submitted: 2016-12-08
• Last Update Posted: 2016-12-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 111

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.

Read More

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/exclusion criteria may apply

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox	Deferasirox	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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
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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇷🇺 St. Petersburg, Russian Federation