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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.

NCT00390858 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2017-03-20

Study results available
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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.

Conditions

  • Transfusional Iron Overload
  • β-thalassemia Major
  • Pediatric Rare Anemia

Interventions

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.

Sponsors & Collaborators

Principal Investigators

  • Dr. Gianluca Forni · Novartis Pharmaceuticals

  • Prof. Renzo Galanello · Novartis Pharmaceuticals

  • Prof. Antonio Piga · Novartis Pharmaceuticals

  • Dr. Yves Bertrand · Novartis Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-09-30
Primary Completion
2008-02-29
Completion
2008-02-29

Countries

  • France
  • Italy

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00390858 on ClinicalTrials.gov