An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

May 24, 2011 updated by: Novartis Pharmaceuticals

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload

A 1-year randomized Phase III core trial (NCT00061750) using deferoxamine as the comparator was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older. Patients who successfully completed this main trial may continue in this extension trial to receive chelation therapy with deferasirox for an additional 4 years.

The objective of this study is to assess the efficacy and long-term safety of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

506

Phase

  • Phase 3

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

  • Argentina
      • Buenos Aires, Argentina
        • Novartis Investigative Site
      • Cordoba, Argentina
        • Novartis Investigative Site
  • Belgium
      • Bruxelles, Belgium
        • Novartis Investigative Site
      • Laken, Belgium
        • Novartis Investigative Site
      • Liege, Belgium
        • Novartis Investigative Site
      • Mons, Belgium
        • Novartis Investigative Site
  • Brazil
      • Campinas, Brazil
        • Novartis Investigative Site
      • Sao Paolo, Brazil
        • Novartis Investigative Site
  • Canada
      • Montreal, Canada
        • Novartis Investigative Site
      • Toronto, Canada
        • Novartis Investigative Site
  • France
      • Creteil, France
        • Novartis Investigative Site
      • Marseille, France
        • Novartis Investigative Site
      • Paris, France
        • Novartis Investigative Site
      • Pierre-Benite, France
        • Novartis Investigative Site
  • Germany
      • Berlin, Germany
        • Novartis Investigative Site
      • Duesseldorf, Germany
        • Novartis Investigative Site
      • Frankfurt, Germany
        • Novartis Investigative Site
      • Hamburg, Germany
        • Novartis Investigative Site
      • Ulm, Germany
        • Novartis Investigative Site
  • Greece
      • Athens, Greece
        • Novartis Investigative Site
      • Ioannina, Greece
        • Novartis Investigative Site
      • Patras, Greece
        • Novartis Investigative Site
      • Thessaloniki, Greece
        • Novartis Investigative Site
  • Italy
      • Brindisi, Italy
        • Novartis Investigative Site
      • Cagliari, Italy
        • Novartis Investigative Site
      • Catania, Italy
        • Novartis Investigative Site
      • Ferrara, Italy
        • Novartis Investigative Site
      • Genova, Italy
        • Novartis Investigative Site
      • Milan, Italy
        • Novartis Investigative Site
      • Monza, Italy
        • Novartis Investigative Site
      • Naples, Italy
        • Novartis Investigative Site
      • Palermo, Italy
        • Novartis Investigative Site
      • Pavia, Italy
        • Novartis Investigative Site
      • Roma, Italy
        • Novartis Investigative Site
      • Sassari, Italy
        • Novartis Investigative Site
      • Siracusa, Italy
        • Novartis Investigative Site
      • Turin, Italy
        • Novartis Investigative Site
  • Tunisia
      • Tunis, Tunisia
        • Novartis Investigative Site
  • Turkey
      • Adana, Turkey
        • Novartis Investigative Site
      • Isparta, Turkey
        • Novartis Investigative Site
      • Istanbul, Turkey
        • Novartis Investigative Site
      • Izmir, Turkey
        • Novartis Investigative Site
  • United Kingdom
      • London, United Kingdom
        • Novartis Investigative Site
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
      • Oakland, California, United States, 94609-1809
        • Children's Hospital and Research Center at Oakland
      • Stanford, California, United States, 94305-5208
        • Stanford Hospital, Division of Oncology
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Children's Memorial Hospital
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Children's Hospital Boston, Dept of Hematology
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-4399
        • Children's Hospital of Philadelphia

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

  • Patients who completed the 12-month core study (NCT00061750)
  • Female patients after menarche and who were sexually active, if they used double-barrier contraception, oral contraceptive plus barrier contraceptive, or had undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
  • Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation

Exclusion criteria

  • Pregnant or breast feeding patients
  • Patients with a history of non-compliance to medical regimens or those considered to be potentially unreliable

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: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Deferasirox
All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.
Drug: Deferasirox
Tablets taken orally once a day.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
Time Frame: up to 5 years
Adverse events results are based on preferred terms with at least 7% of participants in any group.
up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Mean absolute change of LIC from start of Deferasirox (ICL670) treatment to the end of study assessed by liver biopsy. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Mean absolute change in LIC from start of Deferasirox (ICL670) treatment to the end of the study assessed by Superconducting Quantum Interfering Device (SQUID) measurement used as a non-invasive alternative to Biopsy for pediatric participants. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Mean Absolute Change in serum ferritin (ug/L) from start of treatment with Deferasirox (ICL670) to end of study taking into account the therapeutic goal which will either be to maintain iron balance or to induce negative iron balance. End of study taken as the mean of, at most, the last three available results after start of treatment with ICL670.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Measurement of the relative change in percent of potential surrogate marker: Serum Transferrin (g/L) from start of treatment with Deferasirox (ICL670) to end of study.

(Serum Transferrin at the End of Study-Serum Transferrin at Start of ICL670)/Serum Transferrin at Start of ICL670*100.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Measurement of the relative change of potential surrogate markers: Serum Iron (µmol/L) from start of treatment with Deferasirox (ICL670) to end of study.

(Serum Iron at the End of Study-Serum Iron at Start of ICL670)/Serum Iron at Start of ICL670*100.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Measurement of the relative change of potential surrogate marker: Transferrin Saturation (Percent) from start of treatment with Deferasirox (ICL670) to end of study.

(Transferrin Saturation at the End of Study-Tranferrin Saturation at Start of ICL670)/Transferrin Saturation at Start of ICL670*100.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Measurement of median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through biopsy. Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw).
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative change in liver iron content (LIC) as measured by biopsy and calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Measurement of the median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through Superconducting Quantum Interfering Device (SQUID). Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw).
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Relative change in liver iron content (LIC) measured by Superconducting Quantum Interfering Device (SQUID), calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study
Time Frame: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)
Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study.
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

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

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

October 1, 2004

Primary Completion (Actual)

April 1, 2008

Study Registration Dates

First Submitted

September 12, 2005

First Submitted That Met QC Criteria

September 12, 2005

First Posted (Estimate)

September 15, 2005

Study Record Updates

Last Update Posted (Estimate)

May 30, 2011

Last Update Submitted That Met QC Criteria

May 24, 2011

Last Verified

May 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670A0107E1

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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