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

NCT02193074 · ENDEAR Phase 3Terminated

Lead sponsor: Biogen (Industry)
Enrollment: 122 (actual)
Start date: 19 Aug 2014
Primary completion: 21 Nov 2016 (actual)
Study completion: 21 Nov 2016
Last update posted: 17 Feb 2021

Design

Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel
Masking: Quadruple
Arms: 2

Interventions

nusinersen Drug
Sham procedure Procedure

Conditions and primary outcome

Spinal Muscular Atrophy

Primary outcome

Percentage of Motor Milestones Responders (assessed at the later of the Day 183, Day 302, or Day 394 study visits); Time to Death or Permanent Ventilation (Day 91, Day 182, Day 273, Day 364, Day 394)

Posted results on ClinicalTrials.gov

Source: ClinicalTrials.gov results for NCT02193074 (opens in a new tab)· Licence: public-domain

First posted 28 Jul 2017 · Last update posted 17 Feb 2021 · Imported 26 Oct 2026 from ClinicalTrials.gov download 2026-10-25

Primary outcomes with a statistical analysis

Percentage of Motor Milestones Responders
Time frame
assessed at the later of the Day 183, Day 302, or Day 394 study visits
Measure
Number
Unit
percentage of participants
- Control vs NusinersenSuperiority or other
  Estimate
  50.68Difference in percentages
  95% CI, two-sided
  31.81 to 66.48
  p-value
  < 0.0001
  Method
  Fisher Exact
Time to Death or Permanent Ventilation
Time frame
Day 91, Day 182, Day 273, Day 364, Day 394
Measure
Number
Unit
proportion of participants
- Control vs NusinersenSuperiority or other
  Estimate
  Not given
  CI
  Not given
  p-value
  0.0046
  Method
  Log Rank
- Control vs NusinersenSuperiority or other
  Estimate
  0.530Hazard Ratio (HR)
  95% CI, two-sided
  0.3156 to 0.8902
  p-value
  0.0164
  Method
  Cox proportional hazards model

Serious adverse events

Time frame: Screening through Day 394 (± 7 days) or early termination

Participants with serious adverse events per group for ENDEAR, as posted
Group	Affected	At risk
Control	39	41
Nusinersen	61	80

Posted results as ClinicalTrials.gov prints them: primary outcome analyses and serious adverse event totals only. Registry facts, not reviewed by HealthTrade and not investment advice; filing-derived results are listed separately.

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Results from company and journal sources

No editor-confirmed results are on record for this study.

Publications

5 on PubMed

Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy.
Advances in therapy · 2024 Jun
PubMed 38705943 (opens in a new tab)DOI 10.1007/s12325-024-02845-6 (opens in a new tab)
An Integrated Safety Analysis of Infants and Children with Symptomatic Spinal Muscular Atrophy (SMA) Treated with Nusinersen in Seven Clinical Trials.
CNS drugs · 2019 Sep
PubMed 31420846 (opens in a new tab)DOI 10.1007/s40263-019-00656-w (opens in a new tab)
Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1.
Advances in therapy · 2019 May
PubMed 30879249 (opens in a new tab)DOI 10.1007/s12325-019-00923-8 (opens in a new tab)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
The New England journal of medicine · 2017 Nov 2
PubMed 29091570 (opens in a new tab)DOI 10.1056/NEJMoa1702752 (opens in a new tab)
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study.
Lancet (London, England) · 2016 Dec 17
PubMed 27939059 (opens in a new tab)DOI 10.1016/S0140-6736(16)31408-8 (opens in a new tab)

Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.

CT-VCA trial quality

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Data integrity

Compare what the registry, company releases, FDA documents and publications say about this study.

Drugs on HealthTrade

Nusinersen

Source

NCT02193074 on ClinicalTrials.gov (opens in a new tab) Licence: public-domain. Read 25 Oct 2026

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Listing a study does not mean NLM has evaluated it.

Nothing on this page is medical or investment advice.