An Open-Label Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - BN40703

F. Hoffmann-La Roche AG0 个研究点目标入组 26 人开始时间: 2023年9月27日最近更新: 2024年7月22日

适应症

试验速览

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•1. Males and females aged from birth (1 day) to 6 weeks (42 days) of age at the time of first dose (Day 1); a minimum age of 7 days at first dose is required for the first infant to be enrolled, 2. Gestational age of 37-42 weeks for singleton births; gestational age of 34-42 weeks for twins, 3. Body weight = 3rd percentile for age, using appropriate country specific guidelines, 4. Genetic diagnosis of 5q-autosomal recessive SMA, including confirmation of homozygous deletion or compound heterozygosity predictive of loss of function of the SMN1 gene, 5. Absence of clinical signs or symptoms at screening (Day -42 to Day -2) or at baseline (Day -1) that are, in the opinion of the investigator, strongly suggestive of SMA, 6. Receiving adequate nutrition and hydration at the time of screening, in the opinion of the investigator

•1. Concomitant or previous participation in any investigational drug or device study at any time, 2. Concomitant or previous administration of an SMN2-targeting antisense oligonucleotide, SMN2-splicing modifier, or gene therapy either in a clinical study or as part of medical care, 3. Presence of significant concurrent syndromes or diseases, 4. In the opinion of the investigator, inadequate venous or capillary blood access for the study procedures, 5. Requiring invasive ventilation, tracheostomy or awake non-invasive ventilation, 6. Awake hypoxemia (SaO2 < 95%) with or without ventilator support

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