CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 145 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.

Search/NCT04089566
NCT04089566Phase 3Completed
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Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Biogen·interventional·Posted Sep 13, 2019·Updated Jun 5, 2025

In Brief

A Phase 3 clinical trial evaluating Nusinersen for Muscular Atrophy, Spinal. Completed, enrolled 145 participants across 45 sites in 18 countries.

Detailed Summary

The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C). The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMuscular Atrophy, Spinal
CountriesBrazil, Canada, Chile, China, Colombia, Estonia, Germany, Hungary, Italy, Japan, Lebanon, Mexico, Poland, Russia, Saudi Arabia, Spain, Taiwan, United States
Collaborators--

Timeline

Phase 3CompletedFinished
2020202120222023202420252026
First PostedSep 13, 2019
Enrollment StartMar 26, 2020
Primary CompletionFeb 21, 2024
Study CompletionMay 30, 2024
TodayJul 2, 2026
Enrollment to primary: 3.9 yearsPosted 6.8 years ago

Interventions

Nusinersendrug

Administered as specified in the treatment arm