NCT02704273已完成不适用
INCEPTUS: A Prospective, Non-Interventional Clinical Assessment Study in X Linked Myotubular Myopathy (XLMTM) Subjects Aged 3 Years and Younger
Astellas Gene Therapies8 个研究点 分布在 5 个国家目标入组 34 人开始时间: 2016年7月最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 34
- 试验地点
- 8
- 主要终点
- Characterize the disease course and natural history of XLMTM using respiratory assessment of strength as measured by PImax
研究概览
简要总结
This is a pre-Phase 1 prospective, non interventional clinical assessment study to evaluate XLMTM subjects aged 3 years and younger. Many of these clinically relevant measures have not yet been routinely assessed in this population and may provide important insight on the natural history of XLMTM and for future evaluation of potential therapies.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 3 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Subject has a diagnosis of XLMTM resulting from a confirmed mutation in the MTM1 gene
- •Subject is male
- •Subject is aged less than 4 years
- •Subject requires some mechanical ventilatory support (eg, ranging from 24 hours per day full time mechanical ventilation, to noninvasive support such as continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP] during sleeping hours)
- •Access to subject's medical records
- •Signed informed consent by the parent(s) or legally authorized representative(s) (LAR) (when applicable)
- •Subject and parent(s) or LAR are willing and able to comply with study visits and study procedures
排除标准
- •Subject is participating in an interventional study designed to treat XLMTM
- •Subject born <35 weeks gestation who is still not to term as per corrected age
- •Subject has a clinically important condition, or life-threatening disease other than XLMTM, in the opinion of the investigator
- •Subject has received pyridostigmine or any medication to treat XLMTM within 30 days of enrollment
结局指标
主要结局
Characterize the disease course and natural history of XLMTM using respiratory assessment of strength as measured by PImax
时间窗: 3 months to 2 years
Characterize the disease course and natural history of XLMTM using neuromuscular assessments, as measured by CHOP INTEND/MFM-20
时间窗: 3 months to 2 years
次要结局
- Characterize quality of life as measured by PedsQL(3 months to 2 years)
研究者
研究点 (8)
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