A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 40
- 试验地点
- 21
- 主要终点
- The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
研究概览
简要总结
A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adults Male Subjects with Cerebral Adrenoleukodystrophy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subject is male and aged ≥18 years.
- •Subject has progressive cALD, defined as GdE+ brain lesions.
- •Subjects for whom HSCT is not recommended by the investigator or subject is not willing to undergo HSCT.
- •Subject has a Loes score ≥0.5 and ≤12 at Screening.
- •Subject does not have major functional disability in the Major Functional Disabilities-Neurological Function Score (MFD-NFS), except for "wheelchair bound" or "total incontinence", which will be allowed as these are considered expected symptoms of AMN in the time course of the disease
- •Subject does not have major cognitive impairment which would impair his ability to take part in the study as determined by the investigator at screening.
排除标准
- •Subject who had previous bone marrow transplantation (HSCT) or treatment with ex-vivo gene therapy (eli-Cel).
- •Subject has known type 1 or type 2 diabetes.
- •Subject has known hypersensitivity or intolerance to pioglitazone or any other thiazolidinedione.
- •Subject is taking or has taken honokiol, pioglitazone, or other thiazolidinediones within 3 months prior to Screening.
- •Subject with current participation in another interventional clinical study or within 1 month prior to Screening.
- •Subject with other medical, neuropsychiatric or social conditions that, in the opinion of the investigator, are likely to adversely affect the risk-benefit of study participation, interfere with study compliance, or confound the study results.
研究组 & 干预措施
Placebo
Placebo
干预措施: Placebo (Drug)
Leriglitazone
Leriglitazone Treatment
干预措施: Leriglitazone (Drug)
结局指标
主要结局
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
时间窗: Interim analysis 1 (at 18 months of treatment)
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
时间窗: Interim analysis 2 (at 27 months of treatment)
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
时间窗: Final analysis (at 36 months of treatment)
The primary endpoint will be the time to death or the subject becoming bedridden with a requirement for permanent ventilatory support, wichever comes earlier, in subjects treated with leriglitazone compared to placebo.
次要结局
- Change from Baseline in Loes Score(Interim analysis 2 (at 27 months of treatment))
- Change from Baseline in Loes Score(Final analysis (at 36 months of treatment))
- Change from Baseline in Loes Score.(Interim analysis 1 (at 18 months of treatment))