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.

Novartis Pharmaceuticals40 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Transfusional Iron Overload β-thalassemia Major Pediatric Rare Anemia

Interventions

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.

Eligibility

Sex: ALLMin age: 1 YearMax age: 18 Years

Medical Language ↔ Plain English

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.

Locations (4)

Novartis Investigative Site

Lyon, France

Novartis Investigative Site

Cagliari, Italy

Novartis Investigative Site

Genova, Italy

Novartis Investigative Site

Torino, Italy

Outcomes

Primary Outcomes

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.

Time frame: 4 year extension + core 1 year

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)

Time frame: Baseline of Core Study to End of Extension Study, up to 5 years.

Secondary Outcomes

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.

Time frame: Baseline of Core Study to End of Extension Study, up to 5 years

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.

Time frame: Baseline of Core Study to Extension 18 months, up to 2.5 years.