NCT04528706进行中（未招募）2 期
An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
Minoryx Therapeutics, S.L.5 个研究点 分布在 4 个国家目标入组 13 人开始时间: 2019年9月13日最近更新:
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）
- 发起方
- 入组人数
- 13
- 试验地点
- 5
- 主要终点
- evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.
研究概览
简要总结
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
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
- 2 Years 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Males aged ≥2 and ≤12 years with a diagnosis of X-linked ALD
- •White matter involvement as determined by cerebral MRI lesions without Gd enhancement at baseline (Population 1), or with Gd enhancement at baseline (Population 2).
- •Major Functional Disabilities (MFD) score of
- •Baseline Loes score >0 and ≤10
- •Gadolinium Intensity Score >2
排除标准
- •Previous HSCT
- •Too far progressed inflammatory brain lesions
研究组 & 干预措施
MIN-102
Experimental
干预措施: MIN-102 (Drug)
结局指标
主要结局
evaluate whether MIN-102 can halt disease progression of cALD if administered prior to hematopoietic stem-cell transplantation (HSCT), as determined by serial clinical and MRI investigations in pediatric subjects.
时间窗: 6 months- 2 years
"arrested disease" will be assessed at 24 weeks and at 96 weeks
时间窗: at 24 weeks and 96 weeks
arrested disease is defined using: Cahnge in NFS from Baseline, Free of MFD and lack of lesion progression on MRI
次要结局
未报告次要终点
研究者
研究点 (5)
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