NCT02386553

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 0 Weeks – 6 Weeks
• Healthy volunteers: Not accepted

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.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Nusinersen	Solution for intrathecal injection

Timeline

• Start date: 2015-05-18
• Primary completion: 2024-12-17
• Completion: 2024-12-17
• First posted: 2015-03-12
• Last updated: 2025-10-20
• Results posted: 2025-10-20

Locations

21 sites across 7 countries: United States, Australia, Germany, Italy, Qatar, Taiwan, Turkey (Türkiye)