An Open Label Extension Study of PTG-300 In Non-Transfusion Depenent (NTD) and Trasfusion-Dependent (TD) B-Thalassemia Subjects
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 16
- 主要终点
- Proportion of subjects with side effects and severity of side effects will be tabulated
研究概览
简要总结
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
详细描述
The PTG-300-03 study will study beta thalassemia patients on PTG-300 for two years. The dose and dose frequency for each subject enrolling in the PTG-300-03 study will be based on the last dose and frequency received by the subject in the previous study PTG-300-02 and the subject's response at that dose. Patients dose will be increased in a manner similar to that used on PTG-300-02 study. The maximum PTG-300 dose allowed is 80 mg/week.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •NTD and TD β-thalassemia subjects who completed Week 12 and Week 16 respectively in Study PTG-300-02.
排除标准
- •Subjects who discontinued prematurely from study 300-02 (before Week 12 in NTD and Week 16 in TD).
研究组 & 干预措施
Interventions
PTG-300
干预措施: PTG-300 (Drug)
结局指标
主要结局
Proportion of subjects with side effects and severity of side effects will be tabulated
时间窗: Over two year Period after receiving PTG-300
the long-term safety and tolerability of PTG-300 in Beta Thalassemia.
次要结局
未报告次要终点