MedTrace Logo

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

NCT02193074 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 122

Last updated 2021-02-17

Study results available
· View outcomes & findings →

Summary

The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset with infantile-onset spinal muscular atrophy (SMA). The secondary objective of the study is to examine the safety and tolerability of nusinersen administered intrathecally to participants with infantile-onset SMA.

Conditions

Interventions

DRUG

nusinersen

Administered by intrathecal (IT) injection as specified in the treatment arm.

PROCEDURE

Sham procedure

Small needle prick on the lower back at the location where the IT injection is normally made

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Max Age
210 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-08-19
Primary Completion
2016-11-21
Completion
2016-11-21

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • France
  • Germany
  • Italy
  • Japan
  • South Korea
  • Spain
  • Sweden
  • Turkey (Türkiye)
  • United Kingdom

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 NCT02193074 on ClinicalTrials.gov