An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naive, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy (277SM302 (STELLAR-1))

未提供0 个研究点目标入组 30 人开始时间: 待定

适应症

试验速览

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•=<42 days of age at first dose of salanersen.
•Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
•Two or three copies of the survival motor neuron 2 (SMN2) gene.
•Ulnar compound muscle action potential (CMAP) amplitude >= 2 millivolt (mV) at Screening and Day 1 predose.
•Body weight >=3rd percentile for age based on World Health Organization (WHO) Child Growth Standards at the time of informed consent.

•Any clinical signs or symptoms at Screening or Day 1 predose that are, in the opinion of the Investigator, strongly suggestive of SMA.
•Areflexia on neurologic examination at biceps, knee, or ankle at Screening or Day 1 Predose.
•Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or respiratory support, or for altitudes >1000 meters (m), oxygen saturation of <92% awake or asleep without any supplemental oxygen or respiratory support).
•Diagnosis of neonatal respiratory distress syndrome necessitating surfactant replacement therapy or invasive ventilatory support.
•Any reason, anatomical or otherwise (including hematology/coagulation laboratory results), that presents increased risk of complication from the LP procedures or safety assessments.
•Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi [OA], and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.

发起方

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