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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.

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.
 
ClinicalTrials.gov Identifier: NCT01250951
Recruitment Status : Completed
First Posted : December 1, 2010
Last Update Posted : December 12, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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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
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Study Type : Interventional  (Clinical Trial)
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 : December 2009
Actual Primary Completion Date : July 2011
Actual Study Completion Date : September 2011

Resource links provided by the National Library of Medicine

Drug Information available for: Deferasirox

Arms and Interventions
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Arm Intervention/treatment
Experimental: Deferasirox Drug: Deferasirox
Other Name: ICL670


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
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
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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
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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
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Additional Information:

Publications of Results:
Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. Epub 2009 Dec 8. Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. Epub 2009 Nov 30.

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01250951    
Other Study ID Numbers: CICL670ARU01
First Posted: December 1, 2010    Key Record Dates
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:
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Preleukemia
Anemia
Myelodysplastic Syndromes
Thalassemia
Iron Overload
Syndrome
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
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action