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.

Phase 2

Completed

• Conditions: Transfusional Iron Overload; β-thalassemia Major; Pediatric Rare Anemia
• Interventions: Drug: Deferasirox

• Registration Number: NCT00390858
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2003-09
• Study First Submitted: 2006-10-18
• Study First Submitted QC: 2006-10-19
• Study First Posted: 2006-10-20
• Primary Completion: 2008-02
• Study Completion: 2008-02
• Results First Submitted: 2010-12-21
• Results First Submitted QC: 2011-07-20
• Results First Posted: 2011-08-15
• Status Verified: 2017-02
• Last Update Submitted: 2017-02-12
• Last Update Posted: 2017-03-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox	Deferasirox	Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.

Primary Outcome Measures

Name	Time	Method
Participants With Adverse Events by Primary System Organ Class (SOC)	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)	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

Name	Time	Method
Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)	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	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.

Trial Locations

Locations (1)

Novartis Investigative Site; 🇮🇹 Torino, Italy