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.

Transfusional Iron Overload Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00390858
• Other study ID #: CICL670A0106E1
• Status: Completed
• Phase: Phase 2
• First received: October 18, 2006
• Last updated: July 20, 2011
• Start date: September 2003
• Est. completion date: February 2008

Study information

• Verified date: July 2011
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Agenzia Italiana del FarmacoFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: February 2008
• Est. primary completion date: February 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 18 Years

Eligibility

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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta-Thalassemia
• Iron Overload
• Pediatric Rare Anemia
• ß-thalassemia Major
• Thalassemia
• Transfusional Iron Overload

Intervention

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.

Locations

Country	Name	City	State
France	Novartis Investigative Site	Lyon
Italy	Novartis Investigative Site	Cagliari
Italy	Novartis Investigative Site	Genova
Italy	Novartis Investigative Site	Torino

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

France,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	4 year extension + core 1 year	No
Primary	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)	Baseline of Core Study to End of Extension Study, up to 5 years.	No
Secondary	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.	Baseline of Core Study to End of Extension Study, up to 5 years	No
Secondary	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.	Baseline of Core Study to Extension 18 months, up to 2.5 years.	No
Secondary	Relative Change in Serum Transferrin Level	Serum Levels were drawn at Baseline of the Core Study and up to 18 months in the Extension Study. Serum was analyzed for transferrin levels measured as grams per Liter. Relative change (%) in serum transferrin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in transferrin level from Baseline/Baseline level) x 100.	Baseline of Core Study to Extension Study 18 months , up to 2.5 years	No