A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
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| ClinicalTrials.gov Identifier: NCT00390858 |
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Recruitment Status :
Completed
First Posted : October 20, 2006
Results First Posted : August 15, 2011
Last Update Posted : March 20, 2017
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Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
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Brief Summary:
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Transfusional Iron Overload β-thalassemia Major Pediatric Rare Anemia | Drug: Deferasirox | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 40 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-thalassemia Major. |
| Study Start Date : | September 2003 |
| Actual Primary Completion Date : | February 2008 |
| Actual Study Completion Date : | February 2008 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Beta thalassemia
Drug Information available for:
Deferasirox
| Arm | Intervention/treatment |
|---|---|
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Experimental: Deferasirox
Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.
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Drug: Deferasirox
Deferasirox in children from 1 to 18 years old was given orally once daily, 30 minutes prior to breakfast. An initial daily dose of 10 mg/kg was used during the 1-year core study. In this 4-year extension study dose modifications of ± 5 or 10 mg/kg were based on safety parameters and on increasing or decreasing Liver Iron Concentration (LIC), and serum ferritin. Deferasirox was available as 125 mg, 250 mg and 500 mg tablets.
Other Name: ICL670 |
Primary Outcome Measures :
- Participants With Adverse Events by Primary System Organ Class (SOC) [ Time Frame: 4 year extension + core 1 year ]Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product.
- Change in Liver Iron Concentration (LIC) [ Time Frame: Baseline of Core Study to End of Extension Study, up to 5 years. ]Change in Liver Iron Concentration [LIC] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw)
Secondary Outcome Measures :
- Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) [ Time Frame: Baseline of Core Study to End of Extension Study, up to 5 years ]Total Iron Body Elimination (TBIE) Rate [mg/kg/Day] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results.
- Relative Change in Serum Ferritin Level [ Time Frame: Baseline of Core Study to Extension 18 months, up to 2.5 years. ]Serum levels were drawn at the baseline of the Core Study up to 18 months of the Extension Study. Levels were analyzed for serum ferritin measured in micrograms per Liter. Relative change (%) in serum ferritin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in ferritin level from Baseline/Baseline level) x 100.
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| Ages Eligible for Study: | 1 Year to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Completion of the planned 12-month core trial, (NCT00390858).
- Female patients who have reached menarche and who were sexually active were to use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
- Written informed consent obtained from the patient, and/or from the parent or legal guardian in accordance with the national legislation.
Exclusion Criteria:
- Pregnant or breast feeding patients
- Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable.
Other protocol-defined exclusion criteria may apply.
Information from the National Library of Medicine
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): NCT00390858
Locations
| France | |
| Novartis Investigative Site | |
| Lyon, France | |
| Italy | |
| Novartis Investigative Site | |
| Cagliari, Italy | |
| Novartis Investigative Site | |
| Genova, Italy | |
| Novartis Investigative Site | |
| Torino, Italy | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Dr. Gianluca Forni | Novartis Pharmaceuticals | |
| Study Director: | Prof. Renzo Galanello | Novartis Pharmaceuticals | |
| Study Director: | Prof. Antonio Piga | Novartis Pharmaceuticals | |
| Study Director: | Dr. Yves Bertrand | Novartis Pharmaceuticals |
| Responsible Party: | Novartis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT00390858 |
| Other Study ID Numbers: |
CICL670A0106E1 |
| First Posted: | October 20, 2006 Key Record Dates |
| Results First Posted: | August 15, 2011 |
| Last Update Posted: | March 20, 2017 |
| Last Verified: | February 2017 |
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
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β-thalassemia major iron overload deferasirox pediatric rare anemia |
Additional relevant MeSH terms:
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Thalassemia beta-Thalassemia Iron Overload Anemia Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies |
Genetic Diseases, Inborn Iron Metabolism Disorders Metabolic Diseases Deferasirox Iron Chelating Agents Chelating Agents Sequestering Agents Molecular Mechanisms of Pharmacological Action |