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

NCT02386553 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2025-10-20

Study results available
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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

Interventions

DRUG

Nusinersen

Solution for intrathecal injection

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
NA
Purpose
PREVENTION
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Weeks
Max Age
6 Weeks
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-18
Primary Completion
2024-12-17
Completion
2024-12-17

Countries

  • United States
  • Australia
  • Germany
  • Italy
  • Qatar
  • Taiwan
  • Turkey (Türkiye)

Study Locations

More Related Trials

Entities

Diseases
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 NCT02386553 on ClinicalTrials.gov