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

NCT04089566 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 145

Last updated 2025-06-05

Study results available
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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

DRUG

Nusinersen

Administered as specified in the treatment arm

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SEQUENTIAL

Eligibility

Min Age
7 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-03-26
Primary Completion
2024-02-21
Completion
2024-05-30
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Canada
  • Chile
  • China
  • Colombia
  • Estonia
  • Germany
  • Hungary
  • Italy
  • Japan
  • Lebanon
  • Mexico
  • Poland
  • Russia
  • Saudi Arabia
  • Spain
  • Taiwan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04089566 on ClinicalTrials.gov