Safety and Efficacy of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
- Conditions
- Iron Overload
- Interventions
- Drug: Desferasirox
- Registration Number
- NCT01546415
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 64
-
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:
- AA: Clinically diagnosed AA with bone marrow confirmation of the diagnosis
-
Age ≥ 18 years, male or female
-
Written informed consent obtained from patient prior to any screening procedures.
- Patients with serum creatinine > ULN or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥ 1.0 mg/mg in a non-first void urine sample at baseline. If serum creatinine is found to be > ULN or UPCR is found to be ≥ 1 mg/mg the test can be repeated after 1 month.
- Creatinine Clearance <40 ml/min
- Patients with other than AA transfusion-dependent underlying illnesses
- Patients with a previous history of clinically relevant ocular and/or auditory toxicity related to iron chelation
- Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:
- history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding
- history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
- history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
- history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN
- history of urinary obstruction or difficulty in voiding
Other protocol-defined inclusion/exclusion criteria may apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Desferasirox Desferasirox -
- Primary Outcome Measures
Name Time Method 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. Every 4 Weeks for 52 weeks of treatment Difference in serum ferritin from baseline vs. 52 weeks of treatment.
- Secondary Outcome Measures
Name Time Method Correlation between serum ferritin (SF) and transferrin saturation (TFS) Every 4 Weeks for 52 weeks of treatment Study will compare SF with TFS level to find degree of relationship measured by correlation.
Number of dispensed, used, partially used or unused packages of study medication to assess drug usage compliance Every 4 Weeks for 52 weeks of treatment Correlation between dose adjustment (increase or decrease) regimens and transfusional burden Every 3 months for 52 Weeks of treatment Evaluation of the relationship between dose adjustment regimens (as dictated by both efficacy and safety parameters) and transfusional burden will be undertaken.
Number of patients with adverse events, serious adverse events and death Every 4 weeks for 52 Weeks of treatment
Trial Locations
- Locations (1)
Novartis Investigative Site
🇨🇳Tianjin, China