Status:

COMPLETED

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

Lead Sponsor:

Novartis Pharmaceuticals

Conditions:

Non-transfusion Dependent Thalassemia

Eligibility:

All Genders

10+ years

Phase:

PHASE2

Brief Summary

CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload. Patients will be treated either with active treatmen...

Eligibility Criteria

Inclusion

  • Core
  • Male or female aged ≥ 10 years with non-transfusion dependent syndromes, not requiring transfusion within 6 months prior to study start. Note: there was a local country amendment for Greece only to change the age specific inclusion criteria to ≥ 18 years old
  • Liver iron concentration ≥ 5 mg/g dry weight measured by Magnetic resonance imaging (MRI) before study start
  • Serum ferritin \>300 ng/mL at screening
  • Core

Exclusion

  • Hemoglobin S (HbS)-variants of thalassemia syndromes
  • Anticipated regular transfusion program during the study. Patients having a sporadic transfusion (e.g. in case of infection) throughout the study course will not be excluded
  • Any blood transfusion 6 months prior to study start
  • Creatinine clearance ≤ 60 mL/min at screening
  • Serum creatinine above the upper limit of normal at both screening visits
  • Significant proteinuria as indicated by a urine protein/urine creatinine ratio \> 1.0 mg/mg
  • Alanine aminotransferase (ALT) of \> 5 x the upper limit of normal at both screening visits
  • Concomitant therapy with hydroxyurea, erythropoietin, butyrate
  • History of deferasirox treatment
  • Pediatric patients: a patient's weight of below 20 kg
  • Extension Inclusion Criteria:
  • Patients who completed the core CICL670A2209 clinical trial
  • Written informed consent obtained prior entry to one year extension study CICL670A2209
  • Extension

Key Trial Info

Start Date :

November 1 2008

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

June 1 2012

Estimated Enrollment :

166 Patients enrolled

Trial Details

Trial ID

NCT00873041

Start Date

November 1 2008

End Date

June 1 2012

Last Update

July 9 2013

Active Locations (21)

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Page 1 of 6 (21 locations)

1

Children's Hospital & Research Center Oakland

Oakland, California, United States, 94609-1809

2

Children's Memorial Hospital/Division of Hematology/Oncology

Chicago, Illinois, United States, 60614-3394

3

New York Presbyterian Hospital/Weill Medical College of Cornell University

New York, New York, United States, 10021

4

Novartis Investigative Site

Athens, Greece