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.

December 8, 2016 updated by: Novartis Pharmaceuticals

1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients 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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

111

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Russian Federation
      • Moscow, Russian Federation
        • Novartis Investigative Site
      • Saint-Petersburg, Russian Federation
        • Novartis Investigative Site
      • St. Petersburg, Russian Federation
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Deferasirox
Drug: Deferasirox
Other Names:
  • ICL670

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Baseline assessment is followed by monthly assessments for up to 1 year

Secondary Outcome Measures

Outcome Measure
Time Frame
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.
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).
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
From the start of study up to 1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2009

Primary Completion (Actual)

July 1, 2011

Study Completion (Actual)

September 1, 2011

Study Registration Dates

First Submitted

November 11, 2010

First Submitted That Met QC Criteria

November 29, 2010

First Posted (Estimate)

December 1, 2010

Study Record Updates

Last Update Posted (Estimate)

December 12, 2016

Last Update Submitted That Met QC Criteria

December 8, 2016

Last Verified

December 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670ARU01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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