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

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00171210
First received: September 12, 2005
Last updated: May 24, 2011
Last verified: May 2011
  Purpose

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.


Condition Intervention Phase
Transfusional Iron Overload in β-thalassemia
Drug: Deferasirox
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]
    Adverse events results are based on preferred terms with at least 7% of participants in any group.


Secondary Outcome Measures:
  • 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) ] [ Designated as safety issue: No ]
    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).

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

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

  • 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) ] [ Designated as safety issue: Yes ]

    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.


  • 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) ] [ Designated as safety issue: Yes ]

    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.


  • 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) ] [ Designated as safety issue: Yes ]

    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.


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

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

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

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

  • 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) ] [ Designated as safety issue: No ]
    Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study.


Enrollment: 506
Study Start Date: October 2004
Primary Completion Date: April 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox
All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.
Drug: Deferasirox
Tablets taken orally once a day.

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00171210

  Show 49 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00171210     History of Changes
Other Study ID Numbers: CICL670A0107E1
Study First Received: September 12, 2005
Results First Received: December 14, 2010
Last Updated: May 24, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
β-thalassemia
iron overload
deferasirox

Additional relevant MeSH terms:
Beta-Thalassemia
Iron Overload
Thalassemia
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Chelating Agents
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Sequestering Agents

ClinicalTrials.gov processed this record on October 20, 2014