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 | Completed Phase 2 Results

• 1 other identifier: CICL670A0106E1
• Study Type: interventional
• Target: 40
• Locations: 2 countries
• Sites: 4

Brief 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

Keywords

β-thalassemia major; iron overload; deferasirox; pediatric rare anemia

Outcome Measures

Primary Outcomes (2)

• 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

• 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.

Secondary Outcomes (2)

• Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)

  Baseline of Core Study to End of Extension Study, up to 5 years

• Relative Change in Serum Ferritin Level

  Baseline of Core Study to Extension 18 months, up to 2.5 years.

Study Arms (1)

Deferasirox

EXPERIMENTAL

Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.

Drug: Deferasirox

Interventions

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.

Also known as: ICL670

Deferasirox

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (4)

Novartis Investigative Site

Lyon, France

Location

Novartis Investigative Site

Cagliari, Italy

Location

Novartis Investigative Site

Genova, Italy

Location

Novartis Investigative Site

Torino, Italy

Location

MeSH Terms

Conditions

Iron Overload

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

862-778-8300

Study Officials

• 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

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 18, 2006
• First Posted: October 20, 2006
• Study Start: September 1, 2003
• Primary Completion: February 1, 2008
• Study Completion: February 1, 2008
• Last Updated: March 20, 2017
• Results First Posted: August 15, 2011

Record last verified: 2017-02

Locations

IT (3); FR (1)