An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 17
- 试验地点
- 5
- 主要终点
- Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
研究概览
简要总结
This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD).
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
- •Completed both the single ascending dose (SAD) and multiple ascending dose (MAD) phases of predecessor study HALO-DMD-01
- •Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01
- •Ability to provide written informed consent
- •Ambulatory or non-ambulatory
排除标准
- •Recent, substantial change in use of cardiac medications or medications affecting muscle function
- •Clinically significant major disease, not related to DMD
- •Significantly compromised cardio-respiratory function
- •History of severe allergic or anaphylactic reactions
- •Prior treatment with another investigational product in past 6 months
- •Inability to undergo magnetic resonance imaging (MRI)
- •Current drug or alcohol abuse or prior treatment for abuse
研究组 & 干预措施
Cohort 1: HT-100 tablet, Dose 1
• Multiple dose administration: Dose 1
干预措施: HT-100 (Drug)
Cohort 2: HT-100 tablet, Dose 2
• Multiple dose administration: Dose 2
干预措施: HT-100 (Drug)
Cohort 3: HT-100 tablet, Dose 3
• Multiple dose administration: Dose 3
干预措施: HT-100 (Drug)
Cohort 4: HT-100 tablet, Dose 4
• Multiple dose administration: Dose 4
干预措施: HT-100 (Drug)
Cohort 5: HT-100 tablet, Dose 5
• Multiple dose administration: Dose 5
干预措施: HT-100 (Drug)
结局指标
主要结局
Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
时间窗: Months 2, 4, 6, 7
* Target Safety profile by review of adverse events (AEs) * Physical examination findings * Clinical laboratory test results * Other diagnostic testing
次要结局
- Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.(Months 4, 6, 7)
- Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.(Months 4, 6)