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NCT Number: NCT01250951

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.

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

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Key information

Conditions

Myelodysplastic Syndrome Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Bone Marrow Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Iron Metabolism Disorders Iron Overload Metabolic Diseases Myelodysplastic Syndromes Nutritional and Metabolic Diseases Thalassemia

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Novartis Investigative Site, Moscow, Russia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Deferasirox

Drug

Other names: ICL670

Primary outcomes

  1. changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade

    Time frame: Baseline assessment is followed by monthly assessments for up to 1 year

Secondary outcomes

  1. changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination

    Time frame: Baseline assessment is followed by monthly assessments for up to 1 year.

  2. 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

    Time frame: at baseline and 1 at year (at the end of study).

  3. 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.

    Time frame: From the start of study up to 1 year

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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.

Important dates

Study start
2009
Primary completion
2011
Study completion
2011
First posted
Dec 1, 2010
Registry last updated
Dec 12, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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