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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00390858
Other study ID # CICL670A0106E1
Secondary ID
Status Completed
Phase Phase 2
First received October 18, 2006
Last updated July 20, 2011
Start date September 2003
Est. completion date February 2008

Study information

Verified date July 2011
Source Novartis
Contact n/a
Is FDA regulated No
Health authority Italy: Agenzia Italiana del FarmacoFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)United States: Food and Drug Administration
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Completed
Enrollment 40
Est. completion date February 2008
Est. primary completion date February 2008
Accepts healthy volunteers No
Gender Both
Age group 1 Year to 18 Years
Eligibility 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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

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.

Locations

Country Name City State
France Novartis Investigative Site Lyon
Italy Novartis Investigative Site Cagliari
Italy Novartis Investigative Site Genova
Italy Novartis Investigative Site Torino

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

France,  Italy, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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. 4 year extension + core 1 year No
Primary 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) Baseline of Core Study to End of Extension Study, up to 5 years. No
Secondary 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. Baseline of Core Study to End of Extension Study, up to 5 years No
Secondary 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. Baseline of Core Study to Extension 18 months, up to 2.5 years. No
Secondary Relative Change in Serum Transferrin Level 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. Baseline of Core Study to Extension Study 18 months , up to 2.5 years No
See also
  Status Clinical Trial Phase
Completed NCT00560820 - Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload Phase 1
Completed NCT01039636 - Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload Phase 1
Completed NCT00600938 - Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload Phase 2
Completed NCT00673608 - Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload Phase 4
Withdrawn NCT01927913 - Treatment of Iron Overload Requiring Chelation Therapy Phase 2
Completed NCT01241357 - High-Tc Susceptometer to Monitor Transfusional Iron Overload Phase 2
Completed NCT01838291 - Active Drug Surveillance Program of Ferriprox Use N/A
Completed NCT01044186 - A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity Phase 2
Completed NCT00379483 - Extension Study of Iron Chelation Therapy With Deferasirox in Patients With Transfusional Iron Overload Phase 2
Terminated NCT01326845 - Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study Phase 4
Completed NCT01186419 - Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload Phase 2
Terminated NCT01363908 - Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload Phase 2