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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 has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01250951
First Posted: December 1, 2010
Last Update Posted: December 12, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
  Purpose
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.

Condition Intervention Phase
Myelodysplastic Syndrome Thalassemia Drug: Deferasirox Phase 4

Study Type: Interventional
Study Design: 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.

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

Primary Outcome Measures:
  • 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 Outcome Measures:
  • 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. ]
  • 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). ]
  • 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 ]

Enrollment: 111
Study Start Date: December 2009
Study Completion Date: September 2011
Primary Completion Date: July 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox Drug: Deferasirox
Other Name: ICL670

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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/exclusion criteria may apply

  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01250951


Locations
Russian Federation
Novartis Investigative Site
Moscow, Russian Federation
Novartis Investigative Site
Saint-Petersburg, Russian Federation
Novartis Investigative Site
St. Petersburg, Russian Federation
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Additional Information:
Publications:
Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01250951     History of Changes
Other Study ID Numbers: CICL670ARU01
First Submitted: November 11, 2010
First Posted: December 1, 2010
Last Update Posted: December 12, 2016
Last Verified: December 2011

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Iron Overload
hemotranfusion
deferasirox
ferritin
Rare anemia
Transfusional Iron Overload

Additional relevant MeSH terms:
Syndrome
Anemia
Myelodysplastic Syndromes
Preleukemia
Thalassemia
Iron Overload
Disease
Pathologic Processes
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Neoplasms
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Iron
Deferasirox
Trace Elements
Micronutrients
Growth Substances
Physiological Effects of Drugs
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action