Under 210 Days, any sex, with Spinal Muscular Atrophy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Motor Milestones RespondersPrimary· 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,
Group
Value
95% CI
Control
0
Nusinersen
51
Time to Death or Permanent VentilationPrimary· 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.
By Day 91 (13 weeks/3 months)
Group
Value
95% CI
Control
0.268
Nusinersen
0.240
By Day 182 (26 weeks/6 months)
Group
Value
95% CI
Control
0.605
Nusinersen
0.294
By Day 273 (39 weeks/9 months)
Group
Value
95% CI
Control
0.702
Nusinersen
0.404
By Day 364 (52 weeks/12 months)
Group
Value
95% CI
Control
0.735
Nusinersen
0.447
By Day 394 (13 months)
Group
Value
95% CI
Control
0.735
Nusinersen
0.447
Percentage of Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) RespondersSecondary· 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.
Group
Value
95% CI
Control
3
Nusinersen
71
Summary of Time to DeathSecondary· 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.
by Day 91
Group
Value
95% CI
Control
0.195
Nusinersen
0.101
by Day 182
Group
Value
95% CI
Control
0.348
Nusinersen
0.141
by Day 273
Group
Value
95% CI
Control
0.382
Nusinersen
0.173
by Day 364
Group
Value
95% CI
Control
0.419
Nusinersen
0.173
by Day 394
Group
Value
95% CI
Control
0.419
Nusinersen
0.173
Percentage of Participants Not Requiring Permanent VentilationSecondary· Up to Day 394
Group
Value
95% CI
Control
68
Nusinersen
77
Percentage of Compound Muscular Action Potential (CMAP) RespondersSecondary· 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.
Group
Value
95% CI
Control
5
Nusinersen
36
Time to Death or Permanent Ventilation in the Subgroup of Participants Below the Study Median Disease DurationSecondary· 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.
by Day 91
Group
Value
95% CI
Control
0.238
Nusinersen
0.128
by Day 182
Group
Value
95% CI
Control
0.546
Nusinersen
0.128
by Day 273
Group
Value
95% CI
Control
0.697
Nusinersen
0.228
by Day 364
Group
Value
95% CI
Control
0.773
Nusinersen
0.271
by Day 394
Group
Value
95% CI
Control
0.773
Nusinersen
0.271
Time to Death or Permanent Ventilation in the Subgroup of Participants Above the Study Median Disease DurationSecondary· 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.
by Day 91
Group
Value
95% CI
Control
0.300
Nusinersen
0.350
by Day 182
Group
Value
95% CI
Control
0.670
Nusinersen
0.462
by Day 273
Group
Value
95% CI
Control
0.725
Nusinersen
0.584
by Day 364
Group
Value
95% CI
Control
0.725
Nusinersen
0.625
by Day 394
Group
Value
95% CI
Control
0.725
Nusinersen
0.625
Number of Participants Experiencing Adverse Events (AEs), Serious AEs (SAEs) and Discontinuations Due to AEsSecondary· 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 abili
Any event
Group
Value
95% CI
Control
40
Nusinersen
77
Moderate or severe event
Group
Value
95% CI
Control
39
Nusinersen
70
Severe event
Group
Value
95% CI
Control
33
Nusinersen
45
Possibly related or related event
Group
Value
95% CI
Control
6
Nusinersen
9
Related event
Group
Value
95% CI
Control
0
Nusinersen
0
Serious event
Group
Value
95% CI
Control
39
Nusinersen
61
Related serious event
Group
Value
95% CI
Control
0
Nusinersen
0
Treatment discontinuation due to an event
Group
Value
95% CI
Control
16
Nusinersen
13
Number of Participants With AEs Corresponding to Changes in Hematology ValuesSecondary· up to Day 394 (± 7 days) or early termination
Anemia
Group
Value
95% CI
Control
1
Nusinersen
1
Neutrophil count increased
Group
Value
95% CI
Control
0
Nusinersen
1
Leukocytosis
Group
Value
95% CI
Control
1
Nusinersen
0
Number of Participants With AEs Corresponding to Changes in Blood Chemistry ValuesSecondary· up to Day 394 (± 7 days) or early termination
Blood potassium decreased
Group
Value
95% CI
Control
0
Nusinersen
2
Liver function test abnormal
Group
Value
95% CI
Control
0
Nusinersen
1
Alanine aminotransferase increased
Group
Value
95% CI
Control
0
Nusinersen
1
Aspartate aminotransferase increased
Group
Value
95% CI
Control
0
Nusinersen
1
Blood chloride decreased
Group
Value
95% CI
Control
0
Nusinersen
1
Blood iron decreased
Group
Value
95% CI
Control
0
Nusinersen
1
Blood sodium decreased
Group
Value
95% CI
Control
0
Nusinersen
1
C-reactive protein increased
Group
Value
95% CI
Control
1
Nusinersen
2
Number of Participants Meeting Selected Vital Sign Criteria Post-BaselineSecondary· up to Day 394 (± 7 days) or early termination
Systolic blood pressure <90 mmHg
Group
Value
95% CI
Control
36
Nusinersen
74
Systolic blood pressure >140 mmHg
Group
Value
95% CI
Control
4
Nusinersen
4
Systolic blood pressure >160 mmHg
Group
Value
95% CI
Control
0
Nusinersen
0
Diastolic blood pressure <50 mmHg
Group
Value
95% CI
Control
26
Nusinersen
71
Diastolic blood pressure >90 mmHg
Group
Value
95% CI
Control
13
Nusinersen
12
Diastolic blood pressure >100 mmHg
Group
Value
95% CI
Control
3
Nusinersen
0
Pulse rate <60 bpm
Group
Value
95% CI
Control
0
Nusinersen
0
Pulse rate >100 bpm
Group
Value
95% CI
Control
41
Nusinersen
80
Adverse events — posted to ClinicalTrials.gov
Time frame: Screening through Day 394 (± 7 days) or early termination.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03878030 — Effect of Nusinersen on Adults With Spinal Muscular Atrophy
· completed
NCT04591678 — Adults With SMA Treated With Nusinersen
· completed
NCT02594124 — A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Inve
· Phase 3
· completed
NCT02052791 — An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SM
· Phase 1
· completed
NCT01839656 — A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular
· Phase 2
· completed
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Currently open trials in the same condition.
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· NA
· recruiting
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· recruiting
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· recruiting
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· Phase 1, PHASE2
· recruiting
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· NA
· recruiting
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Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Biogen
Last refreshed: 17 February 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02193074.