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Completed

NCT Number: NCT00390858

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.

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.

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Key information

Conditions

Transfusional Iron Overload Iron Metabolism Disorders Iron Overload Metabolic Diseases Nutritional and Metabolic Diseases Pediatric Rare Anemia β-thalassemia Major

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, Lyon, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Deferasirox

Drug

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 names: ICL670

Primary outcomes

  1. 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.

  2. 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 outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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.

Important dates

Study start
2003
Primary completion
2008
Study completion
2008
First posted
Oct 20, 2006
Registry last updated
Mar 20, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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