Trial · NCT02193074

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

NCT02193074, Phase 3, Terminated, Biogen; results first posted 2017-07-28

3 sourcestrials-2026-11-12-0.5.0

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ConductScience Library. A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy. Trials, record NCT02193074. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02193074

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
NCT number: NCT02193074
Status: Terminated
Phases: Phase 3
Enrollment: 122 participants, actual
Lead sponsor: Biogen
Conditions: Spinal Muscular Atrophy
Interventions: nusinersen, Sham procedure
First posted: 2014-07-17
Last updated: 2021-02-17
Other registries' IDs: eudract:2013-004422-29
Results first posted: 2017-07-28

Source ClinicalTrials.gov

Registered outcomes14

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percentage of Motor Milestones Responders	assessed at the later of the Day 183, Day 302, or Day 394 study visits	The definition of a motor milestones responder was based on improvement in the motor milestones categories in Section 2 of the Hammersmith Infant Neurological Examination (HINE), with the exclusion of voluntary grasp, as follows: (i) subject demonstrates ≥ 2-point increase in the motor milestones category of ability to kick or achievement of maximal score on that category (touching toes), or a 1-point increase in the motor milestones category of head control, rolling, sitting, crawling, standing, or walking, and (ii) among the motor milestone categories, with the exclusion of voluntary grasp, there are more categories where there is improvement as defined in (i) than worsening. (For the category of ability to kick, worsening is defined as ≥ 2-point decrease or decrease to the lowest possible score of no kicking. For the other categories, worsening is defined as ≥ 1-point decrease.) The lowest possible score for the HINE is 0 (zero), and the highest possible score for the HINE is 28.
Primary	Time to Death or Permanent Ventilation	Day 91, Day 182, Day 273, Day 364, Day 394	Estimated proportion of participants who died or required permanent ventilation by a given study day, based on the Kaplan-Meier product-limit method. Time to death or permanent ventilation was defined as either tracheostomy or ≥ 16 hours ventilation/day continuously for \> 21 days in the absence of an acute reversible event. This endpoint was adjudicated by a blinded, independent group of experienced clinicians, the Event Adjudication Committee (EAC), based on review of clinical study data and supporting information. Results are based on all available data.
Secondary	Percentage of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Responders	assessed at Baseline and the later of the Day 183, Day 302, or Day 394 study visits	A participants was considered a CHOP-INTEND responder if the change from baseline in CHOP-INTEND total score is ≥ 4 points based on assessment at the later of the Day 183, Day 302, or Day 394 study visits. CHOP-INTEND tests includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). Total scores range from 0 to 64, with higher scores indicating better movement functioning. Results are based on all available data.
Secondary	Summary of Time to Death	Day 91, Day 182, Day 273, Day 364, Day 394	Estimated proportion of participants who died by given duration thresholds, based on the Kaplan-Meier product-limit method.
Secondary	Percentage of Participants Not Requiring Permanent Ventilation	Up to Day 394
Secondary	Percentage of Compound Muscular Action Potential (CMAP) Responders	assessed at the later of the Day 183, Day 302, or Day 394 study visits	CMAP is an electrophysiological technique that can be used to determine the approximate number of motor neurons in a muscle or group of muscles. A participant was defined as a CMAP responder if the CMAP amplitude at the peroneal nerve was increasing to or maintained at ≥ 1 mV (comparing to the baseline) based on assessment at the later of the Day 183, Day 302, or Day 394 study visits. Results are based on all available data.
Secondary	Time to Death or Permanent Ventilation in the Subgroup of Participants Below the Study Median Disease Duration	Day 91, Day 182, Day 273, Day 364, Day 394	Estimated proportion of participants who died or required permanent ventilation (EAC-adjudicated events) among participants below the study median disease duration (13.1 weeks), by given duration thresholds, based on the Kaplan-Meier product-limit method.
Secondary	Time to Death or Permanent Ventilation in the Subgroup of Participants Above the Study Median Disease Duration	Day 91, Day 182, Day 273, Day 364, Day 394	Estimated proportion of participants who died or required permanent ventilation (EAC-adjudicated events) among participants above the study median disease duration (13.1 weeks), by given duration thresholds, based on the Kaplan-Meier product-limit method.
Secondary	Number of Participants Experiencing Adverse Events (AEs), Serious AEs (SAEs) and Discontinuations Due to AEs	Screening through Day 394 (± 7 days) or early termination	AE: any unfavorable and unintended sign, symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. SAE: any AE that in the view of either the Investigator or Sponsor, meets any of the following criteria: results in death; is life threatening: that is, poses an immediate risk of death at the time of the event; requires in-patient hospitalization or prolongation of existing hospitalization; results in a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions; results in congenital anomaly or birth defect in the offspring of the participant (whether male or female); is an important medical event in the opinion of the Investigator or Sponsor.
Secondary	Number of Participants With AEs Corresponding to Changes in Hematology Values	up to Day 394 (± 7 days) or early termination

1 to 10 of 14Source ClinicalTrials.gov

See all 14 registered outcomes

Outcomes14

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percentage of Motor Milestones Responders	assessed at the later of the Day 183, Day 302, or Day 394 study visits	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure with Day 183, Day 302, or Day 394 data; the last available assessment was used. (Participants who died or withdrew from the study were counted as non-responders and were included in the denominator for the calculation of the percentages.)	percentage of participants	NUMBER
PRIMARY	Time to Death or Permanent Ventilation	Day 91, Day 182, Day 273, Day 364, Day 394	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure and who died or required permanent ventilation.	proportion of participants	NUMBER
SECONDARY	Percentage of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Responders	assessed at Baseline and the later of the Day 183, Day 302, or Day 394 study visits	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure with Day 183, Day 302, or Day 394 data; the last available assessment was used. (Participants who died or withdrew from the study were counted as non-responders and were included in the denominator for the calculation of the percentages.)	percentage of participants	NUMBER
SECONDARY	Summary of Time to Death	Day 91, Day 182, Day 273, Day 364, Day 394	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure and who died. Results are based on all available data.	proportion of particiants	NUMBER
SECONDARY	Percentage of Participants Not Requiring Permanent Ventilation	Up to Day 394	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure.	percentage of participants	NUMBER
SECONDARY	Percentage of Compound Muscular Action Potential (CMAP) Responders	assessed at the later of the Day 183, Day 302, or Day 394 study visits	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure with Day 183, Day 302, or Day 394 data; the last available assessment was used. (Participants who died or withdrew from the study were counted as non-responders and were included in the denominator for the calculation of the percentages.)	percentage of participants	NUMBER
SECONDARY	Time to Death or Permanent Ventilation in the Subgroup of Participants Below the Study Median Disease Duration	Day 91, Day 182, Day 273, Day 364, Day 394	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure and were below the study median disease duration.	proportion of participants	NUMBER
SECONDARY	Time to Death or Permanent Ventilation in the Subgroup of Participants Above the Study Median Disease Duration	Day 91, Day 182, Day 273, Day 364, Day 394	Intent-to-treat population: all randomized participants who received ≥ 1 dose of study drug/sham procedure and were above the study median disease duration.	proportion of participants	NUMBER
SECONDARY	Number of Participants Experiencing Adverse Events (AEs), Serious AEs (SAEs) and Discontinuations Due to AEs	Screening through Day 394 (± 7 days) or early termination	Safety Population: all subjects who received ≥ 1 dose of study drug/sham procedure.	participants	NUMBER
SECONDARY	Number of Participants With AEs Corresponding to Changes in Hematology Values	up to Day 394 (± 7 days) or early termination	Safety Population: all subjects who received ≥ 1 dose of study drug/sham procedure.	participants	NUMBER

1 to 10 of 14Source ClinicalTrials.gov

See all 14 outcomes

Results by group130

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Units
Percentage of Motor Milestones Responders	Control		0	percentage of participants
Percentage of Motor Milestones Responders	Nusinersen		51	percentage of participants
Time to Death or Permanent Ventilation	Control	By Day 91 (13 weeks/3 months)	0.268	proportion of participants
Time to Death or Permanent Ventilation	Nusinersen	By Day 91 (13 weeks/3 months)	0.240	proportion of participants
Time to Death or Permanent Ventilation	Control	By Day 182 (26 weeks/6 months)	0.605	proportion of participants
Time to Death or Permanent Ventilation	Nusinersen	By Day 182 (26 weeks/6 months)	0.294	proportion of participants
Time to Death or Permanent Ventilation	Control	By Day 273 (39 weeks/9 months)	0.702	proportion of participants
Time to Death or Permanent Ventilation	Nusinersen	By Day 273 (39 weeks/9 months)	0.404	proportion of participants
Time to Death or Permanent Ventilation	Control	By Day 364 (52 weeks/12 months)	0.735	proportion of participants
Time to Death or Permanent Ventilation	Nusinersen	By Day 364 (52 weeks/12 months)	0.447	proportion of participants

1 to 10 of 130Source ClinicalTrials.gov

See all 130 results by group

Participant flow10

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Control	41
Overall Study	STARTED	Nusinersen	80
Overall Study	Completed During Follow-Up Period	Control	11
Overall Study	Completed During Follow-Up Period	Nusinersen	26
Overall Study	Completed Due to Early Study Termination	Control	13
Overall Study	Completed Due to Early Study Termination	Nusinersen	39
Overall Study	COMPLETED	Control	24
Overall Study	COMPLETED	Nusinersen	65
Overall Study	NOT COMPLETED	Control	17
Overall Study	NOT COMPLETED	Nusinersen	15

Source ClinicalTrials.gov

Adverse events286

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Bradycardia	Cardiac disorders	Control	3	41
other	Bradycardia	Cardiac disorders	Nusinersen	4	80
other	Tachycardia	Cardiac disorders	Control	5	41
other	Tachycardia	Cardiac disorders	Nusinersen	7	80
other	Constipation	Gastrointestinal disorders	Control	9	41
other	Constipation	Gastrointestinal disorders	Nusinersen	28	80
other	Diarrhoea	Gastrointestinal disorders	Control	7	41
other	Diarrhoea	Gastrointestinal disorders	Nusinersen	11	80
other	Dysphagia	Gastrointestinal disorders	Control	9	41
other	Dysphagia	Gastrointestinal disorders	Nusinersen	7	80

1 to 10 of 286Source ClinicalTrials.gov

See all 286 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Control
Total, all-cause mortality	Nusinersen
Total, other adverse events	Control	35	41
Total, other adverse events	Nusinersen	70	80
Total, serious adverse events	Control	39	41
Total, serious adverse events	Nusinersen	61	80

Source ClinicalTrials.gov

Papers5

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
27939059	DERIVED	PubMed
29091570	DERIVED	PubMed
30879249	DERIVED	PubMed
31420846	DERIVED	PubMed
38705943	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Nusinersen vs Control: Number of Participants With Clinically Significant Changes From Baseline in Urinalysis Values	Trial comparisons
Nusinersen vs Control: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT02193074

GET https://conductscience.org/api/trials/v1/NCT02193074/results?page=2

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