clinical trial · NCT02386553
A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Biogen·phase2·completed·n = 25
Spinal Muscular AtrophyNusinersen
brief summary
The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.
started
May 18, 2015
primary completion
Dec 17, 2024
completion
Dec 17, 2024
last updated
Oct 20, 2025
official title
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol