Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)

Brief Title

Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)

Official Title

1-year Extension to CICL670A2402 an Open-label, Multi-center Trial of the Efficacy and Safety of Long-term Treatment With Deferasirox (10 to 20 mg/kg/Day) in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2- Year Duration)

Brief Summary

      To allow patients treated with deferasirox in the core study to continue iron chelation
      therapy for 2 years or until the drug became locally commercially available. To evaluate the
      long-term safety and efficacy of deferasirox by measuring treatment success, change in liver
      iron content (LIC) and change in serum ferritin levels. Safety was mainly assessed by
      incidence of adverse events (AEs)and clinically significant lab parameters.
    

Detailed Description

      Iron accumulation is an inevitable consequence of chronic blood transfusions and results in
      serious complications in the absence of chelation treatment to remove excess iron.
      Deferasirox (Exjade, ICL670) is an oral chelator with high iron-binding potency and
      selectivity. This extension study aimed at collecting efficacy and safety data during 2 years
      of treatment with deferasirox in the extension study or until deferasirox became commercially
      available in the countries where the centers were located, whichever came first. The
      population comprised of β-thalassemia patients with transfusional hemosiderosis who could not
      be satisfactorily treated with deferoxamine or deferiprone.
    

Study Phase

Phase 4

Study Type

Interventional


Primary Outcome

Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age

Secondary Outcome

 Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study

Condition

Beta-thalassemia Major

Intervention

Deferasirox

Study Arms / Comparison Groups

 Deferasirox
Description:  Deferasirox was given orally once daily (10 to 20 mg/kg) to participants 2 years and older based on participant's body weight.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

233

Start Date

June 2005

Completion Date

May 2008

Primary Completion Date

May 2008

Eligibility Criteria

        Inclusion Criteria:

          -  Patients completing the planned 12-month core study (NCT00171171)

          -  Female patients who have reached menarche and who are sexually active must use an
             effective method of contraception, or must have undergone clinically documented total
             hysterectomy and/or ovariectomy, or tubal ligation

          -  Written informed consent obtained from the patient and/or legal guardian on the
             patient's behalf in accordance with the national legislation

        Exclusion Criteria:

          -  Pregnant or breast feeding patients.

          -  Patients being considered by the investigator potentially unreliable and/or not
             cooperative with regard to the core study protocol, or the planned extension protocol
      

Gender

All

Ages

2 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Novartis Pharmaceuticals, , 

Location Countries

Egypt

Location Countries

Egypt

Administrative Informations


NCT ID

NCT00171301

Organization ID

CICL670A2402E1


Responsible Party

Sponsor

Study Sponsor

Novartis Pharmaceuticals


Study Sponsor

Novartis Pharmaceuticals, Study Director, Novartis Pharmaceuticals


Verification Date

August 2011