Back to resultsA 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.
Primary Purpose
Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
About this trial
This is an interventional treatment trial for Transfusional Iron Overload focused on measuring β-thalassemia major, iron overload, deferasirox, pediatric rare anemia
Eligibility 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.
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Deferasirox
Arm Description
Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.
Outcomes
Primary Outcome Measures
Participants With Adverse Events by Primary System Organ Class (SOC)
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)
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)
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
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.
Full Information
NCT ID
NCT00390858
First Posted
October 18, 2006
Last Updated
February 12, 2017
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00390858
Brief Title
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.
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 Type
Interventional
2. Study Status
Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
September 2003 (undefined)
Primary Completion Date
February 2008 (Actual)
Study Completion Date
February 2008 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
Keywords
β-thalassemia major, iron overload, deferasirox, pediatric rare anemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
40 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Deferasirox
Arm Type
Experimental
Arm Description
Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Other Intervention Name(s)
ICL670
Intervention Description
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.
Primary Outcome Measure Information:
Title
Participants With Adverse Events by Primary System Organ Class (SOC)
Description
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.
Time Frame
4 year extension + core 1 year
Title
Change in Liver Iron Concentration (LIC)
Description
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)
Time Frame
Baseline of Core Study to End of Extension Study, up to 5 years.
Secondary Outcome Measure Information:
Title
Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)
Description
Total Iron Body Elimination (TBIE) Rate [mg/kg/Day] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results.
Time Frame
Baseline of Core Study to End of Extension Study, up to 5 years
Title
Relative Change in Serum Ferritin Level
Description
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.
Time Frame
Baseline of Core Study to Extension 18 months, up to 2.5 years.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dr. Gianluca Forni
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Prof. Renzo Galanello
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Prof. Antonio Piga
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Dr. Yves Bertrand
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Lyon
Country
France
Facility Name
Novartis Investigative Site
City
Cagliari
Country
Italy
Facility Name
Novartis Investigative Site
City
Genova
Country
Italy
Facility Name
Novartis Investigative Site
City
Torino
Country
Italy
12. IPD Sharing Statement
Learn more about this trial
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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