EUCTR2017-004087-35-IT进行中（未招募）1 期
A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT
AveXis, Inc.0 个研究点目标入组 44 人开始时间: 2018年4月23日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 44
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •All patients:
- •Age =6 weeks (=42 days) at time of dose
- •Ability to tolerate thin liquids as demonstrated through a formal bedside swallowing test
- •Compound muscle action potential (CMAP) =2 mV at Baseline
- •Gestational age of 35 to 42 weeks
- •Genetic diagnosis as described below, obtained from an acceptable newborn or pre-natal screening test method
- •Patients with 2 copies of SMN2
- •Patients with pre-symptomatic SMA Type 1 as determined by the following features:
- •Bi-allelic deletion of SMN1
- •2 copies of SMN2
- •Patients with 3 copies of SMN2
- •Patients with pre-symptomatic SMA Type 2 as determined by the following features:
- •Bi-allelic deletion of SMN1
- •3 copies of SMN2
- •Patients with 4 copies of SMN2
- •Patients predominantly with pre-symptomatic SMA Type 3 as determined by the following features:
- •Bi-allelic deletion of SMN1
- •4 copies of SMN2
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 44
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Weight at screening visit <2 kg
- •Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or respiratory support) at the screening visit
- •Any clinical signs or symptoms at screening or immediately prior to dosing that are, in the opinion of the Investigator, strongly suggestive of SMA (e.g., tongue fasciculation, hypotonia, areflexia)
- •Tracheostomy or current prophylactic use or requirement of non invasive ventilatory support at any time and for any duration prior to screening or during the screening period
- •Patients with signs of aspiration/inability to tolerate non thickened liquids based on a formal swallowing test performed as part of screening or patients receiving any non-oral feeding method
- •Treatment with an investigational or commercial product, including nusinersen, given for the treatment of SMA. This includes any history of gene therapy, prior antisense oligonucleotide treatment, or cell transplantation.
- •Patients whose weight-for-age is below the third percentile based on World Health Organization (WHO) Child Growth Standards
- •Serious non respiratory tract illness requiring systemic treatment and/or hospitalization within 2 weeks prior to screening
- •Upper or lower respiratory infection requiring medical attention, medical intervention, or increase in supportive care of any manner within 4 weeks prior to dosing
- •Previous, planned or expected major surgical procedure including scoliosis repair surgery/procedure during the study assessment period
- •Concomitant use of any of the following: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, immunosuppressive therapy within 4 weeks prior to gene replacement therapy (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, IV immunoglobulin, rituximab)
- •Anti AAV9 antibody titer >1:50 as determined by Enzyme linked Immunosorbent Assay (ELISA) binding immunoassay
研究者
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