An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload
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| ClinicalTrials.gov Identifier: NCT00171210 |
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Recruitment Status :
Completed
First Posted : September 15, 2005
Results First Posted : May 2, 2011
Last Update Posted : May 30, 2011
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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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Transfusional Iron Overload in β-thalassemia | Drug: Deferasirox | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 506 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (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 |
| Study Start Date : | October 2004 |
| Actual Primary Completion Date : | April 2008 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Deferasirox
All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.
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Drug: Deferasirox
Tablets taken orally once a day. |
- 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.
- 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 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) ]Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study.
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| Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00171210
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| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
| Responsible Party: | External Affairs, Novartis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT00171210 |
| Other Study ID Numbers: |
CICL670A0107E1 |
| First Posted: | September 15, 2005 Key Record Dates |
| Results First Posted: | May 2, 2011 |
| Last Update Posted: | May 30, 2011 |
| Last Verified: | May 2011 |
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β-thalassemia iron overload deferasirox |
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Thalassemia beta-Thalassemia Iron Overload Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies |
Genetic Diseases, Inborn Iron Metabolism Disorders Metabolic Diseases Deferasirox Iron Chelating Agents Chelating Agents Sequestering Agents Molecular Mechanisms of Pharmacological Action |