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:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01250951
First received: November 11, 2010
Last updated: December 1, 2011
Last verified: December 2011
  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: 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:

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 ] [ Designated as safety issue: No ]

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. ] [ Designated as safety issue: No ]
  • 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). ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]

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

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: 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:
No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01250951     History of Changes
Other Study ID Numbers: CICL670ARU01
Study First Received: November 11, 2010
Last Updated: December 1, 2011
Health Authority: Russia: Ministry of Health

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

Additional relevant MeSH terms:
Anemia
Myelodysplastic Syndromes
Preleukemia
Thalassemia
Iron Overload
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
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014