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.

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00390858
First received: October 18, 2006
Last updated: July 20, 2011
Last verified: July 2011
  Purpose

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.


Condition Intervention Phase
Transfusional Iron Overload
β-thalassemia Major
Pediatric Rare Anemia
Drug: Deferasirox
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official 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.

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Participants With Adverse Events by Primary System Organ Class (SOC) [ Time Frame: 4 year extension + core 1 year ] [ Designated as safety issue: No ]
    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) [ Time Frame: Baseline of Core Study to End of Extension Study, up to 5 years. ] [ Designated as safety issue: No ]
    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:
  • Total Body Iron Elimination (TBIE) Rate (mg/kg/Day) [ Time Frame: Baseline of Core Study to End of Extension Study, up to 5 years ] [ Designated as safety issue: No ]
    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 [ Time Frame: Baseline of Core Study to Extension 18 months, up to 2.5 years. ] [ Designated as safety issue: No ]
    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.

  • Relative Change in Serum Transferrin Level [ Time Frame: Baseline of Core Study to Extension Study 18 months , up to 2.5 years ] [ Designated as safety issue: No ]
    Serum Levels were drawn at Baseline of the Core Study and up to 18 months in the Extension Study. Serum was analyzed for transferrin levels measured as grams per Liter. Relative change (%) in serum transferrin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in transferrin level from Baseline/Baseline level) x 100.


Enrollment: 40
Study Start Date: September 2003
Study Completion Date: February 2008
Primary Completion Date: February 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox
Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.
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.
Other Name: ICL670

  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00390858

Locations
France
Novartis Investigative Site
Lyon, France
Italy
Novartis Investigative Site
Cagliari, Italy
Novartis Investigative Site
Genova, Italy
Novartis Investigative Site
Torino, Italy
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Dr. Gianluca Forni Novartis Pharmaceuticals
Study Director: Prof. Renzo Galanello Novartis Pharmaceuticals
Study Director: Prof. Antonio Piga Novartis Pharmaceuticals
Study Director: Dr. Yves Bertrand Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: External Affairs, Novartis
ClinicalTrials.gov Identifier: NCT00390858     History of Changes
Other Study ID Numbers: CICL670A0106E1
Study First Received: October 18, 2006
Results First Received: December 21, 2010
Last Updated: July 20, 2011
Health Authority: Italy: Agenzia Italiana del Farmaco
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United States: Food and Drug Administration

Keywords provided by Novartis:
β-thalassemia major
iron overload
deferasirox
pediatric rare anemia

Additional relevant MeSH terms:
Thalassemia
Iron Overload
Beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014