NCT03570931进行中（未招募）2 期
A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 19
- 试验地点
- 2
- 主要终点
- Modified Ashworth Spasticity Scale
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
详细描述
This is a single arm open-label study with a structured observation of INAD patients treated with RT001. Enrolled subjects will undergo observation and testing to determine the effect of RT001 treatment. Fifteen to twenty eligible subjects will be treated with RT001 for long-term evaluation of efficacy, safety, tolerability, and pharmacokinetics.
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
盲法说明
Open Label
入排标准
- 年龄范围
- 18 Months 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female 18 months to 10 years of age
- •Medical history consistent with the symptoms of classic INAD (onset of symptoms between the ages of 6 months and 3 years)
- •Homozygous for PLA2G6 deficiency (variant alleles may be mixed heterozygotes)
- •Must have impairment in at least 2 of the assessed categories at baseline
- •Signed informed consent form (ICF) prior to entry into the study
- •Able to provide the necessary blood samples
排除标准
- •Received treatment with other experimental therapies within the last 30 days prior to the first dose
- •Requiring mechanical ventilation, other than positive air pressure support primarily for mitigation of sleep apnea.
- •Have a life expectancy of less than one year
- •Diagnosis of atypical NAD (ANAD)
- •Unwilling or unable to comply with the requirements of this protocol, including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to return for visits as scheduled
研究组 & 干预措施
RT001
Experimental
RT001, oral, 3.84 g/day
干预措施: RT001 (Drug)
结局指标
主要结局
Modified Ashworth Spasticity Scale
时间窗: 12 months
Change from baseline in the Modified Ashworth spasticity scale.
次要结局
- Progression Free Survival Time(All available data)
- INAD Progression Composite(12 months)
研究者
研究点 (2)
Loading locations...
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