Clinical trial NCT00171171: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Beta-Thalassemia
Hemosiderosis
Keywords: Thalassemia (beta-thal. major)
Transfusional hemosiderosis
Deferasirox
Beta-thalassemia major patients
Unable to be chelated with deferoxamine or deferiprone
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2005-09-15
Enrollment: 252
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Liver Iron Concentration (as measured by biopsy)
timeFrame: at baseline and after 1 yeor of ICL670 treatment
Secondary outcomes:
measure: 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)
timeFrame: after 1 year of ICL670 treatment
measure: Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation
timeFrame: monthly assessments for 1 year
measure: Adverse events
timeFrame: during 1 year ICL670 treatment
measure: hematology and biochemistry, urinalysis
timeFrame: monthly assessments for 1 year ICL670 treatment
measure: ECG and Echocardiography
timeFrame: 6-monthly for 1 year ICL670 treatment
Other outcomes:
Locations:
city: Cairo
country: Egypt
facility: Novartis Investigative Site
city: Beirut
country: Lebanon
facility: Ali Taher
city: Muscat
country: Oman
facility: Novartis Investigative Site
city: Riyadh
country: Saudi Arabia
facility: Novartis Investigative Site
city: Damascus
country: Syrian Arab Republic
facility: Novartis Investigative Site
Study officials:
affiliation: American University of Beirut Medical Center
name: Ali Taher, Ass.Prof.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Taher A, Al Jefri A, Elalfy MS, Al Zir K, Daar S, Rofail D, Baladi JF, Habr D, Kriemler-Krahn U, El-Beshlawy A. Improved treatment satisfaction and convenience with deferasirox in iron-overloaded patients with beta-Thalassemia: Results from the ESCALATOR Trial. Acta Haematol. 2010;123(4):220-5. doi: 10.1159/000313447. Epub 2010 Apr 27.