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

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

Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy
Enrollment: 122
Study Start Date: July 2014
Study Completion Date: November 2016
Primary Completion Date: November 2016 (Final data collection date for primary outcome measure)

Arms:
- Experimental: nusinersen
- Sham Comparator: Sham procedure

Related journal:
- Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.