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A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

NCT02292537 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 126

Last updated 2021-02-17

Study results available
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Summary

The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.

Conditions

Interventions

DRUG

Nusinersen

Administered by intrathecal (IT) lumbar puncture (LP) injection

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

Min Age
2 Years
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-11-24
Primary Completion
2017-02-20
Completion
2017-02-20

Countries

  • United States
  • Canada
  • France
  • Germany
  • Hong Kong
  • Italy
  • Japan
  • South Korea
  • Spain
  • Sweden

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 NCT02292537 on ClinicalTrials.gov