NCT00040677已完成2 期
A Phase II, Multicenter, Twelve-Week, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Range-Finding Study of the Efficacy and Safety of ICA-17043 With or Without Hydroxyurea Therapy in Patients With Sickle Cell Anemia
Icagen1 个研究点 分布在 1 个国家目标入组 90 人开始时间: 2002年2月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 90
- 试验地点
- 1
- 主要终点
- The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
研究概览
简要总结
ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks RBC dehydration. ICA-17043 is expected to inhibit RBC dehydration and thus should prevent or delay the sickling process. By reducing sickled cells, an improvement in anemia, a reduction in painful crises, and ultimately, less end-organ disease is anticipated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Homozygous (HbSS) Sickle Cell Anemia
- •Otherwise healthy (based on medical history, physical examination, 12-lead ECG, and clinical laboratory tests)
- •Patients may be receiving hydroxyurea, but must have been dose stabilized for at least 3 months
- •Patient has a history of at least one acute vaso-occlusive event requiring hospitalization
排除标准
- •Patient participating in a chronic transfusion program
- •Patient having a total hemoglobin of < 4.0 g/dL or > 10.0 g/dL
- •Patient having a HbA > 10%
- •Patient considering undergoing an elective surgery
- •Patient taking prohibited medications such as Epoetin, Warfarin, etc.
- •Patient who has had previous gastrointestinal surgery, except cholecystectomy or appendectomy
- •Patient with significant active cardiovascular, neurologic, endocrine, hepatic, or renal disorders unrelated to sickle cell anemia
研究组 & 干预措施
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
ICA-17043 Low Dose 6 mg/day
Experimental
Active study medication: 100 mg loading dose; 6 mg maintenance dose per day
干预措施: Low Dose ICA-17043 (Drug)
ICA-17043 High Dose 10 mg/day
Experimental
Active study medication: 150 mg loading dose; 10 mg maintenance dose per day
干预措施: High dose ICA-17043 (Drug)
结局指标
主要结局
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
时间窗: 12 Weeks
次要结局
- Changes in other hematologic measurements(12 weeks)
- Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH(12 weeks)
- Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)(12 weeks)
- Rate of painful crises(12 weeks)
- Time to first painful crisis(12 weeks)
- Morbidity of painful crises (maximum morbidity index, derived variable)(12 weeks)
- Pain intensity scores(12 weeks)
- Quality of Life (SF 36)(12 Weeks)
- Health economic data(12 weeks)
- Average plasma concentration(12 weeks)
- Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint(12 weeks)
研究者
研究点 (1)
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