Beta-Thalassemia Clinical Trial
Official title:
A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
| Verified date | November 2016 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 252 |
| Est. completion date | |
| Est. primary completion date | November 2006 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 2 Years and older |
| Eligibility |
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 |
| Country | Name | City | State |
|---|---|---|---|
| Egypt | Novartis Investigative Site | Cairo | |
| Lebanon | Ali Taher | Beirut | |
| Oman | Novartis Investigative Site | Muscat | |
| Saudi Arabia | Novartis Investigative Site | Riyadh | |
| Syrian Arab Republic | Novartis Investigative Site | Damascus |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis |
Egypt, Lebanon, Oman, Saudi Arabia, Syrian Arab Republic,
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 — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Liver Iron Concentration (as measured by biopsy) | at baseline and after 1 yeor of ICL670 treatment | ||
| Secondary | 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 | ||
| Secondary | Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation | monthly assessments for 1 year | ||
| Secondary | Adverse events | during 1 year ICL670 treatment | ||
| Secondary | hematology and biochemistry, urinalysis | monthly assessments for 1 year ICL670 treatment | ||
| Secondary | ECG and Echocardiography | 6-monthly for 1 year ICL670 treatment |
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