A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Brief Title

A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Official Title

A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Brief Summary

      Because patients with beta-thalassemia are unable to actively eliminate iron from the body,
      toxic and eventually lethal levels of iron can accumulate as a result of repeated blood
      transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Liver Iron Concentration (as measured by biopsy)

Secondary Outcome

 Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)

Condition

Beta-Thalassemia

Intervention

deferasirox

Study Arms / Comparison Groups

 Deferasirox
Description:  

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

252

Start Date

May 2004


Primary Completion Date

November 2006

Eligibility Criteria

        Inclusion Criteria:

          -  Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be
             chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to
             documented unacceptable toxicity of DFO or documented poor response to DFO despite
             proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500
             ng/ml and LIC ≥ 2 mg/Fe/g dw liver

          -  Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone

        Exclusion Criteria:

          -  Means levels of ALT > 300 U/L

          -  Serum creatinine above upper limit of normal

          -  Active hepatitis C or chronic hepatitis B receiving specific treatment
      

Gender

All

Ages

2 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Ali Taher, Ass.Prof., , 

Location Countries

Egypt

Location Countries

Egypt

Administrative Informations


NCT ID

NCT00171171

Organization ID

CICL670A2402



Study Sponsor

Novartis


Study Sponsor

Ali Taher, Ass.Prof., Principal Investigator, American University of Beirut Medical Center


Verification Date

November 2016