NL-OMON56788招募中4 期
An open-label, single-center, exploratory study of the safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged >= 5 years. - neoGAA, phase IV
适应症
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 6
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 64（—）
入选标准
- •- Age >= 5 years and =< 50 years.
- •- Childhood or juvenile/young adult onset.
- •- Residing in the Netherlands or Belgium.
- •- Current Enzyme-replacement therapy with alglucosidase alfa >= 2 years (dose
- •regimen 20 or 40 mg/kg bi-weekly).
- •- Confirmed diagnosis: enzyme deficiency in any tissue source / 2 confirmed
- •pathogenic variations in the GAA gene.
- •- Willing and able to adhere to study procedures (incl. patient and/or
- •parent/guardian signed informed consent).
- •- Deterioration in either pulmonary function / 6MWT / muscle strength despite
- •current treatment regimen with alglucosidase alfa.
- •- Disease status:
- •- Measurable pulmonary (dys)function: (F)VC <= 80% predicted (mechanic
- •ventilation during the
- •day or night allowed).
- •- Measurable muscle weakness in proximal and/or distal muscle groups
- •ambulant/wheelchair bound patients allowed).
- •- Measurable functional ability.
排除标准
- •- Age >50 years.
- •- Invasive mechanical ventilation.
- •- No remaining useful functional ability (e.g. (almost) tetraplegic), as
- •decided by the treating physician.
- •- Unmanageable, severe IARs on alglucosidase alfa.
- •- Deterioration due to high levels of anti-alglucosidase alfa antibodies
- •interfering with treatment efficacy.
- •- Female patient of childbearing potential not protected by highly effective
- •contraceptive method of birth control and/or who is unwilling or unable to be
- •tested for pregnancy
研究者
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