At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 25 enrolled
Drug / intervention
Nusinersendrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 2 clinical trial evaluating Nusinersen for Spinal Muscular Atrophy. Completed, enrolled 25 participants across 21 sites in 7 countries.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesAustralia, Germany, Italy, Qatar, Taiwan, Turkey (Türkiye), United States
Collaborators--
Timeline
Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedMar 2015
Enrollment StartMay 2015
Primary CompletionDec 2024
TodayJul 2026
First PostedMar 12, 2015
Enrollment StartMay 18, 2015
Primary CompletionDec 17, 2024
TodayJul 2, 2026
Enrollment to primary: 9.6 yearsPosted 11.3 years ago
Interventions
Nusinersendrug
Solution for intrathecal injection