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

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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00171171

Enrollment

252

Registered

2005-09-15

Start date

2004-05-31

Completion date

Unknown

Last updated

2017-03-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Beta-Thalassemia, Hemosiderosis

Keywords

Thalassemia (beta-thal. major), Transfusional hemosiderosis, Deferasirox, Beta-thalassemia major patients, Unable to be chelated with deferoxamine or deferiprone

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.

Interventions

DRUGdeferasirox

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

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

Secondary

Measure	Time frame
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

Countries

Egypt, Lebanon, Oman, Saudi Arabia, Syria

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026