Clinical Trial: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload
Brief Summary:
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.
Detailed Summary:
Sponsor: Novartis Pharmaceuticals
Current Primary Outcome: 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 ]
Original Primary Outcome:
Current Secondary Outcome:
- 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).
- 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).
- 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.
- 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.
- 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.
- 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.
- 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).
- 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.
- 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).
- 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 Sup
Original Secondary Outcome:
Information By: Novartis
Dates:
Date Received: September 12, 2005
Date Started: October 2004
Date Completion:
Last Updated: May 24, 2011
Last Verified: May 2011
