An Open-label, Dose-escalation, Dose-finding, and Proof-of-concept Trial of SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 90
- 试验地点
- 1
- 主要终点
- To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia
研究概览
简要总结
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
The main questions it aims to answer are:
- How efficient is SP-420 in cleaning iron from the liver?
- How is the safety and tolerability of ascending doses of SP-420?
Participants will:
- Take medication three times weekly
- Attend up to 20 site visits
- Undergo MRI scans
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 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
- 未提供
排除标准
- 未提供
研究组 & 干预措施
3c-3d
SP-420 for 48 weeks
干预措施: SP-420 (Drug)
Cohort 1a-1b
SP-420 for 48 weeks
干预措施: SP-420 (Drug)
Cohort 2a-2b
SP-420 for 48 weeks
干预措施: SP-420 (Drug)
Cohort 3a-3b
SP-420 for 48 weeks
干预措施: SP-420 (Drug)
1c-1d
SP-420 for 48 weeks
干预措施: SP-420 (Drug)
2c-2d
SP-420 for 48 weeks
干预措施: SP-420 (Drug)
结局指标
主要结局
To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia
时间窗: 24 weeks
To assess the safety and tolerability of ascending doses of SP-420 after 12 weeks treatment of subjects with transfusion-dependent low-risk myelodysplastic syndrome
时间窗: 12 weeks
次要结局
- To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia(24 weeks)
- To assess the efficacy of SP-420 on serum (s-) ferritin(up to 48 weeks)
- To assess the efficacy of SP-420 in clearing iron from the liver after 12 and 48 weeks treatment of subjects with transfusion-dependent β-thalassemia(12 and 48 weeks)
- To assess the safety and tolerability of ascending doses of SP-420(48 weeks)