EUCTR2014-002098-12-DE进行中（未招募）1 期
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - NURTURE
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 25
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
- •- Age = 6 weeks at first dose
- •- Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
- •- Genetic documentation of 2 or 3 copies of survival motor neuron 2 (SMN2).
- •- Ulnar Compound muscle action potential (CMAP) = 1 mV at Baseline.
- •- Gestational age of 37 to 42 weeks for singleton births; gestational age of 34 to 42 weeks for twins.
- •- Meet additional study related criteria.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 25
- •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
- •- Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or respiratory support).
- •- Any clinical signs or symptoms at Screening or immediately prior to the first dosing (Day1) that are, in the opinion of the Investigator, strongly suggestive of SMA.
- •- Clinically significant abnormalities in hematology or clinical chemistry parameters.
- •- Treatment with an investigational drug given for the treatment of SMA biological agent, or device. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation.
- •- Meet additional study related criteria.
研究者
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