NCT01546415已完成4 期
A Single Arm, Multicenter, Open Label Study of Desferasirox in Chinese Patients With Iron Overload and Aplastic Anemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 64
- 试验地点
- 1
- 主要终点
- 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.
研究概览
简要总结
The study is designed to collect safety and efficacy of Desferasirox in Chinese patients with Iron Overload and Aplastic Anemia.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
Desferasirox
Experimental
干预措施: Desferasirox (Drug)
结局指标
主要结局
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.
时间窗: Every 4 Weeks for 52 weeks of treatment
Difference in serum ferritin from baseline vs. 52 weeks of treatment.
次要结局
- Correlation between serum ferritin (SF) and transferrin saturation (TFS)(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(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)
- Number of patients with adverse events, serious adverse events and death(Every 4 weeks for 52 Weeks of treatment)
研究者
研究点 (1)
Loading locations...
相似试验
终止
2 期
Deferasirox in Treating Patients With Iron Overload After Undergoing a Donor Stem Cell TransplantIron OverloadMultiple Myeloma and Plasma Cell NeoplasmMyelodysplastic SyndromesBreast CancerOvarian CancerLymphomaLeukemiaNeuroblastomaNCT00602446Masonic Cancer Center, University of Minnesota4
已完成
4 期
Open-Label Single-Arm Pilot Study in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron OverloadIron OverloadNCT01335035Novartis Pharmaceuticals30
已完成
1 期
Study to Investigate the Effect of Deferasirox on the Pharmacokinetics of MidazolamHealthyNCT00427505Novartis22
已完成
2 期
Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional HemosiderosisChronic AnemiaTransfusional HemosiderosisNCT00631163Novartis Pharmaceuticals102
已完成
3 期
Safety, Tolerability, and Efficacy of Deferasirox in MDSMyelodysplastic SyndromesHemosiderosisNCT00469560Gruppo Italiano Malattie EMatologiche dell'Adulto158