Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Brief Title

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Official Title

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload

Brief Summary

      A 1-year randomized Phase II core trial was conducted to investigate the efficacy of
      deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia
      2 years of age and older. Patients who successfully completed the main trial may continue in
      the extension trial to receive chelation therapy with deferasirox for up to 3 years.
      Extension was prolonged to 4 years.

      The objective of this study is to assess the long-term safety and efficacy of deferasirox in
      these patient groups.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths

Secondary Outcome

 The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study

Condition

Anemia

Intervention

Deferasirox

Study Arms / Comparison Groups

 Deferasirox
Description:  Deferasirox daily oral dose between 5-40 mg/kg/day

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

184

Start Date

March 2004

Completion Date

October 2008

Primary Completion Date

October 2008

Eligibility Criteria

        Inclusion Criteria:

          -  Patients completed the planned 12-month core study

          -  Female patients who have reached menarche and who are sexually active must 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 legal guardian on the
             patient's behalf in accordance with the national legislation

        Exclusion Criteria:

          -  Pregnant or breast feeding patients

        Other protocol-defined inclusion/exclusion criteria may apply
      

Gender

All

Ages

2 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Novartis Pharmaceuticals, , 

Location Countries

Belgium

Location Countries

Belgium

Administrative Informations


NCT ID

NCT00303329

Organization ID

CICL670A0108E1



Study Sponsor

Novartis Pharmaceuticals


Study Sponsor

Novartis Pharmaceuticals, Study Director, Novartis Pharmaceuticals


Verification Date

April 2011