A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 17
- 试验地点
- 5
- 主要终点
- Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
研究概览
简要总结
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 20 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Ambulatory or non-ambulatory
- •Diagnosis of DMD with confirmation of minimal to no dystrophin
- •Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen)
排除标准
- •Recent, substantial change in use of cardiac medications or medications affecting muscle function
- •Inability to undergo magnetic resonance imaging (MRI)
- •Significantly compromised cardio-respiratory function
- •Prior treatment with another investigational product in past 6 months
研究组 & 干预措施
Cohort 1: HT-100 tablet, Dose 1
- Single dose administration: Dose 1
- Multiple dose administration: Dose 1
干预措施: HT-100 (Drug)
Cohort 2: HT-100 tablet, Dose 2
- Single dose administration: Dose 2
- Multiple dose administration: Dose 2
干预措施: HT-100 (Drug)
Cohort 3: HT-100 tablet, Dose 3
- Single dose administration: Dose 3
- Multiple dose administration: Dose 3
干预措施: HT-100 (Drug)
Cohort 4a: HT-100 tablet, Dose 4
- Single dose administration: Dose 4
- Multiple dose administration: Dose 4
干预措施: HT-100 (Drug)
Cohort 4b: HT-100 tablet, Dose 5
* Multiple dose administration: Dose 5
干预措施: HT-100 (Drug)
Cohort 5: HT-100 tablet, Dose 6
* Multiple dose administration: Dose 5
干预措施: HT-100 (Drug)
结局指标
主要结局
Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
时间窗: 1 week
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
次要结局
- Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys(1 week)
- Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks(4 weeks)
- Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys(4 weeks)