Transfusional Iron Overload in ß-thalassemia Clinical Trial
Official title:
An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload
| Verified date | May 2011 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | United States: Food and Drug Administration |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 506 |
| Est. completion date | |
| Est. primary completion date | April 2008 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 2 Years and older |
| Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Argentina | Novartis Investigative Site | Buenos Aires | |
| Argentina | Novartis Investigative Site | Cordoba | |
| Belgium | Novartis Investigative Site | Bruxelles | |
| Belgium | Novartis Investigative Site | Laken | |
| Belgium | Novartis Investigative Site | Liege | |
| Belgium | Novartis Investigative Site | Mons | |
| Brazil | Novartis Investigative Site | Campinas | |
| Brazil | Novartis Investigative Site | Sao Paolo | |
| Canada | Novartis Investigative Site | Montreal | |
| Canada | Novartis Investigative Site | Toronto | |
| France | Novartis Investigative Site | Creteil | |
| France | Novartis Investigative Site | Marseille | |
| France | Novartis Investigative Site | Paris | |
| France | Novartis Investigative Site | Pierre-Benite | |
| Germany | Novartis Investigative Site | Berlin | |
| Germany | Novartis Investigative Site | Duesseldorf | |
| Germany | Novartis Investigative Site | Frankfurt | |
| Germany | Novartis Investigative Site | Hamburg | |
| Germany | Novartis Investigative Site | Ulm | |
| Greece | Novartis Investigative Site | Athens | |
| Greece | Novartis Investigative Site | Ioannina | |
| Greece | Novartis Investigative Site | Patras | |
| Greece | Novartis Investigative Site | Thessaloniki | |
| Italy | Novartis Investigative Site | Brindisi | |
| Italy | Novartis Investigative Site | Cagliari | |
| Italy | Novartis Investigative Site | Catania | |
| Italy | Novartis Investigative Site | Ferrara | |
| Italy | Novartis Investigative Site | Genova | |
| Italy | Novartis Investigative Site | Milan | |
| Italy | Novartis Investigative Site | Monza | |
| Italy | Novartis Investigative Site | Naples | |
| Italy | Novartis Investigative Site | Palermo | |
| Italy | Novartis Investigative Site | Pavia | |
| Italy | Novartis Investigative Site | Roma | |
| Italy | Novartis Investigative Site | Sassari | |
| Italy | Novartis Investigative Site | Siracusa | |
| Italy | Novartis Investigative Site | Turin | |
| Tunisia | Novartis Investigative Site | Tunis | |
| Turkey | Novartis Investigative Site | Adana | |
| Turkey | Novartis Investigative Site | Isparta | |
| Turkey | Novartis Investigative Site | Istanbul | |
| Turkey | Novartis Investigative Site | Izmir | |
| United Kingdom | Novartis Investigative Site | London | |
| United States | Children's Hospital Boston, Dept of Hematology | Boston | Massachusetts |
| United States | Children's Memorial Hospital | Chicago | Illinois |
| United States | Children's Hospital Los Angeles | Los Angeles | California |
| United States | Children's Hospital and Research Center at Oakland | Oakland | California |
| United States | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
| United States | Stanford Hospital, Division of Oncology | Stanford | California |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
United States, Argentina, Belgium, Brazil, Canada, France, Germany, Greece, Italy, Tunisia, Turkey, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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. | up to 5 years | Yes |
| Secondary | 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). | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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). | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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. | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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. |
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | Yes |
| Secondary | 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. |
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | Yes |
| Secondary | 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. |
Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | Yes |
| Secondary | 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). | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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. | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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). | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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. | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |
| Secondary | 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. | Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years) | No |