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

Phase 3

Completed

• Conditions: Hemosiderosis; Beta-Thalassemia
• Interventions: Drug: deferasirox

• Registration Number: NCT00171171
• Lead Sponsor: Novartis

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2004-05
• Study First Submitted: 2005-09-12
• Study First Submitted QC: 2005-09-14
• Study First Posted: 2005-09-15
• Primary Completion: 2006-11
• Status Verified: 2016-11
• Last Update Submitted: 2017-02-28
• Last Update Posted: 2017-03-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 252

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox	deferasirox	-

Primary Outcome Measures

Name	Time	Method
Liver Iron Concentration (as measured by biopsy)	at baseline and after 1 yeor of ICL670 treatment

Secondary Outcome Measures

Name	Time	Method
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)	after 1 year of ICL670 treatment
Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation	monthly assessments for 1 year
Adverse events	during 1 year ICL670 treatment
hematology and biochemistry, urinalysis	monthly assessments for 1 year ICL670 treatment
ECG and Echocardiography	6-monthly for 1 year ICL670 treatment

Trial Locations

Locations (2)

Novartis Investigative Site; 🇸🇾 Damascus, Syrian Arab Republic

Ali Taher; 🇱🇧 Beirut, Lebanon