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

Phase 3

Completed

Conditions: Transfusional Iron Overload in β-thalassemia

Interventions: Drug: Deferasirox

Registration Number: NCT00171210

Lead Sponsor: Novartis Pharmaceuticals

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 Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 506

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox	Deferasirox	All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy	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	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	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	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	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	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	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	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	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	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.
Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study	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.

Trial Locations

Locations (7): Children's Hospital and Research Center at Oakland
🇺🇸
Oakland, California, United States
Children's Hospital of Philadelphia
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Philadelphia, Pennsylvania, United States
Novartis Investigative Site
🇬🇧
London, United Kingdom
Stanford Hospital, Division of Oncology
🇺🇸
Stanford, California, United States
Children's Memorial Hospital
🇺🇸
Chicago, Illinois, United States
Children's Hospital Boston, Dept of Hematology
🇺🇸
Boston, Massachusetts, United States
Children's Hospital Los Angeles
🇺🇸
Los Angeles, California, United States