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The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Desferasirox | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Desferasirox | Drug | Initial Dose: 20 mg/kg/d. Dose modification according to protocol. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in serum ferritin from baseline to 52 weeks with desferasirox treatment in Chinese patients with AA presenting with transfusional hemosiderosis and pre-existing serum ferritin levels of ≥ 1000 ng/mL. | Difference in serum ferritin from baseline vs. 52 weeks of treatment. | Every 4 Weeks for 52 weeks of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Correlation between serum ferritin (SF) and transferrin saturation (TFS) | Study will compare SF with TFS level to find degree of relationship measured by correlation. | Every 4 Weeks for 52 weeks of treatment |
| Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance |
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Inclusion Criteria:
Patients presenting with transfusion-related IOL as shown by a SF level of ≥ 1000 ng/mL at start of study
History of transfusion >20 international units or 100 mL/kg of red blood cells
Underlying transfusion-dependent illness:
Age ≥ 18 years, male or female
Written informed consent obtained from patient prior to any screening procedures.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Shanghai | Shanghai Municipality | 200437 | China | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26876245 | Derived | Shi J, Chang H, Zhang L, Shao Y, Nie N, Zhang J, Huang J, Zhang L, Tang X, Quan R, Zheng C, Xiao H, Hu D, Hu L, Liu F, Zhou Y, Zheng Y, Zhang F. [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China]. Zhonghua Xue Ye Xue Za Zhi. 2016 Jan;37(1):1-6. doi: 10.3760/cma.j.issn.0253-2727.2016.01.001. Chinese. |
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| ID | Term |
|---|---|
| D019190 | Iron Overload |
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D019189 | Iron Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D000740 | Anemia |
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| Every 4 Weeks for 52 weeks of treatment |
| Correlation between dose adjustment (increase or decrease) regimens and transfusional burden | Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken. | Every 3 months for 52 Weeks of treatment |
| Number of patients with adverse events, serious adverse events and death | Every 4 weeks for 52 Weeks of treatment |
| Chengdu |
| Sichuan |
| 610041 |
| China |
| Novartis Investigative Site | Tianjin | Tianjin Municipality | 300020 | China |
| Novartis Investigative Site | Beijing | China |
| Novartis Investigative Site | Chengdu | China |
| Novartis Investigative Site | Shanghai | China |
| Novartis Investigative Site | Tianjin | China |
| D006402 |
| Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |