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

Primary Purpose

Transfusional Iron Overload in β-thalassemia

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Deferasirox

About this trial

This is an interventional treatment trial for Transfusional Iron Overload in β-thalassemia focused on measuring β-thalassemia, iron overload, deferasirox

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm Description

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

Outcomes

Primary Outcome Measures

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event

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

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

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

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

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

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

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

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

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

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

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

Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study.

Full Information

NCT ID

NCT00171210

First Posted

September 12, 2005

Last Updated

May 24, 2011

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT00171210

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2011

Overall Recruitment Status

Completed

Study Start Date

October 2004 (undefined)

Primary Completion Date

April 2008 (Actual)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Transfusional Iron Overload in β-thalassemia

Keywords

β-thalassemia, iron overload, deferasirox

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

506 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Deferasirox

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Deferasirox

Intervention Description

Tablets taken orally once a day.

Primary Outcome Measure Information:

Title

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event

Description

Adverse events results are based on preferred terms with at least 7% of participants in any group.

Time Frame

up to 5 years

Secondary Outcome Measure Information:

Title

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy

Description

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).

Time Frame