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

Novartis252 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

252

Conditions

Beta-Thalassemia Hemosiderosis

Interventions

deferasiroxDRUG

Eligibility

Sex: ALLMin age: 2 Years

Medical Language ↔ Plain English

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

Locations (5)

Novartis Investigative Site

Cairo, Egypt

Ali Taher

Beirut, Lebanon

Novartis Investigative Site

Muscat, Oman

Novartis Investigative Site

Riyadh, Saudi Arabia

Novartis Investigative Site

Outcomes

Primary Outcomes

Liver Iron Concentration (as measured by biopsy)

Time frame: at baseline and after 1 yeor of ICL670 treatment

Secondary Outcomes

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)

Time frame: after 1 year of ICL670 treatment

Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation

Time frame: monthly assessments for 1 year

Adverse events

Time frame: during 1 year ICL670 treatment

hematology and biochemistry, urinalysis

Time frame: monthly assessments for 1 year ICL670 treatment

ECG and Echocardiography

Time frame: 6-monthly for 1 year ICL670 treatment

Data from ClinicalTrials.gov

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