NCT04089566

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 145 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 7 Days
• Healthy volunteers: Not accepted

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).

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
DRUG	Nusinersen	Administered as specified in the treatment arm

Timeline

• Start date: 2020-03-26
• Primary completion: 2024-02-21
• Completion: 2024-05-30
• First posted: 2019-09-13
• Last updated: 2025-06-05
• Results posted: 2025-06-05

Locations

45 sites across 18 countries: United States, Brazil, Canada, Chile, China, Colombia, Estonia, Germany, Hungary, Italy, Japan, Lebanon, Mexico, Poland, Russia, Saudi Arabia, Spain, Taiwan

Regulatory

• FDA-regulated drug study