NCT03269045已完成1 期
An Open-label, Single-arm, Phase 2 Study of ORL-1B in Patients With Biotinidase Deficiency
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Orpha Labs
- 入组人数
- 20
- 主要终点
- Improvement in seizure frequency
研究概览
简要总结
An Open-label, Single-arm, Phase 2 Study of Biotin in Patients With Biotinidase Deficiency.
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
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of biotinidase deficiency.
- •Less than 18 years old.
排除标准
- •Diagnosis of any other disease that is not a manifestation of biotinidase deficiency.
研究组 & 干预措施
Treatment with ORL-1B.
Other
Pediatric patients with biotinidase deficiency.
干预措施: ORL-1B (Drug)
结局指标
主要结局
Improvement in seizure frequency
时间窗: 12 months
Statistically significant improvement in seizure frequency after the ORL-1B treatment.
次要结局
未报告次要终点
研究者
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