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

Sponsor

Novartis (Industry)

Overall Status

Completed

CT.gov ID

NCT00171171

Collaborator

(none)

252

Enrollment

Locations

Arm

50.4

Patients Per Site

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Beta-Thalassemia Hemosiderosis	Drug: deferasirox	Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

252 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

May 1, 2004

Actual Primary Completion Date :

Nov 1, 2006

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Deferasirox	Drug: deferasirox

Arm

Intervention/Treatment

Experimental: Deferasirox

Drug: deferasirox

Outcome Measures

Primary Outcome Measures

Liver Iron Concentration (as measured by biopsy) [at baseline and after 1 yeor of ICL670 treatment]

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Novartis Investigative Site	Cairo	Egypt
2	Ali Taher	Beirut	Lebanon	Riad El Solh 1107 2020
3	Novartis Investigative Site	Muscat	Oman
4	Novartis Investigative Site	Riyadh	Saudi Arabia
5	Novartis Investigative Site	Damascus	Syrian Arab Republic