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

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ClinicalTrials.gov Identifier: NCT01839656
Recruitment Status : Completed
First Posted : April 25, 2013
Results First Posted : October 30, 2018
Last Update Posted : October 30, 2018
Sponsor:
Information provided by (Responsible Party):
Biogen

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Spinal Muscular Atrophy
Intervention Drug: nusinersen
Enrollment 21
Recruitment Details  
Pre-assignment Details Of the 23 participants screened, 2 were screening failures. A total of 21 participants enrolled; 1 was withdrawn from the study due to respiratory failure prior to receiving the first dose of study treatment. Twenty participants received at least 1 dose of study treatment and were included in the efficacy, safety, and PK analyses.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261. Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Period Title: Overall Study
Started 5 16
Completed Loading Dose Period 4 15
Completed 2 0
Not Completed 3 16
Reason Not Completed
Withdrawal by Subject             1             1
Adverse event, serious fatal             1             4
Early study closure             0             11
Adverse Event             1             0
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg Total
Hide Arm/Group Description Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261. Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261. Total of all reporting groups
Overall Number of Baseline Participants 4 16 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
<=18 years
4
 100.0%
16
 100.0%
20
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
Female
1
  25.0%
7
  43.8%
8
  40.0%
Male
3
  75.0%
9
  56.3%
12
  60.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
Hispanic or Latino
0
   0.0%
1
   6.3%
1
   5.0%
Not Hispanic or Latino
4
 100.0%
15
  93.8%
19
  95.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
White
3
  75.0%
13
  81.3%
16
  80.0%
Black
0
   0.0%
1
   6.3%
1
   5.0%
Asian
0
   0.0%
1
   6.3%
1
   5.0%
American Indian or Alaskan Native
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Multiple Race
1
  25.0%
0
   0.0%
1
   5.0%
Other
0
   0.0%
1
   6.3%
1
   5.0%
Hammersmith Infant Neurological Examination (HINE) Head Control Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
Unable to maintain head upright
3
  75.0%
13
  81.3%
16
  80.0%
Wobbles
1
  25.0%
2
  12.5%
3
  15.0%
All the time maintained upright
0
   0.0%
1
   6.3%
1
   5.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Sitting Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
Cannot sit
4
 100.0%
15
  93.8%
19
  95.0%
Sits with support at hips
0
   0.0%
1
   6.3%
1
   5.0%
Props
0
   0.0%
0
   0.0%
0
   0.0%
Stable sit
0
   0.0%
0
   0.0%
0
   0.0%
Pivots (rotates)
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Voluntary Grasp Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
No grasp
0
   0.0%
3
  18.8%
3
  15.0%
Uses whole hand
4
 100.0%
13
  81.3%
17
  85.0%
Index finger and thumb but immature grasp
0
   0.0%
0
   0.0%
0
   0.0%
Pincer grasp
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Ability to Kick Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
No kicking
1
  25.0%
5
  31.3%
6
  30.0%
Kick horizontally, legs do not lift
3
  75.0%
10
  62.5%
13
  65.0%
Upward (vertically)
0
   0.0%
1
   6.3%
1
   5.0%
Touches leg
0
   0.0%
0
   0.0%
0
   0.0%
Touches toes
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Rolling Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
No rolling
4
 100.0%
15
  93.8%
19
  95.0%
Rolling side to side
0
   0.0%
0
   0.0%
0
   0.0%
Prone to supine
0
   0.0%
1
   6.3%
1
   5.0%
Supine to prone
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Crawling Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
Does not lift head
4
 100.0%
14
  87.5%
18
  90.0%
On elbow
0
   0.0%
1
   6.3%
1
   5.0%
On outstretched hand
0
   0.0%
1
   6.3%
1
   5.0%
Crawling flat on abdomen
0
   0.0%
0
   0.0%
0
   0.0%
Crawling on hands and knees
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Standing Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
Does not support weight
4
 100.0%
15
  93.8%
19
  95.0%
Supports weight
0
   0.0%
1
   6.3%
1
   5.0%
Stands with support
0
   0.0%
0
   0.0%
0
   0.0%
Stands unaided
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
HINE Walking Motor Milestones at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
No walking
4
 100.0%
15
  93.8%
19
  95.0%
Bouncing
0
   0.0%
1
   6.3%
1
   5.0%
Cruising (walks holding on)
0
   0.0%
0
   0.0%
0
   0.0%
Walking independently
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
Distribution of Total Scores of the CHOP-INTEND Scale at Baseline   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 16 participants 20 participants
0 to 9
0
   0.0%
0
   0.0%
0
   0.0%
10 to 19
0
   0.0%
2
  12.5%
2
  10.0%
20 to 29
3
  75.0%
6
  37.5%
9
  45.0%
30 to 39
1
  25.0%
6
  37.5%
7
  35.0%
40 to 49
0
   0.0%
1
   6.3%
1
   5.0%
50 to 59
0
   0.0%
0
   0.0%
0
   0.0%
60 to 64
0
   0.0%
1
   6.3%
1
   5.0%
[1]
Measure Description: The Children's Hospital of Philadelphia Infant Test for Neuromuscular Disease (CHOP-INTEND) test includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0 (worst) to 4 (best). Total scores range from 0 to 64, with higher scores indicating better movement functioning. Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
Mean CHOP INTEND Total Scores at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 4 participants 16 participants 20 participants
27  (5) 30  (12) 30  (11)
[1]
Measure Description: The CHOP-INTEND test includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0 (worst) to 4 (best). Total scores range from 0 to 64, with higher scores indicating better movement functioning. Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
Mean Compound Muscle Action Potential (CMAP) Amplitude of the Peroneal Nerve at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  Millivolts (mV)
Number Analyzed 4 participants 16 participants 20 participants
0.673  (0.501) 0.518  (0.664) 0.549  (0.626)
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
Mean CMAP Amplitude of the Ulnar Nerve at Baseline   [1] 
Mean (Standard Deviation)
Unit of measure:  mV
Number Analyzed 4 participants 16 participants 20 participants
0.372  (0.177) 0.532  (0.878) 0.501  (0.786)
[1]
Measure Description: Baseline was defined as the last non-missing value prior to the first dose of nusinersen.
1.Primary Outcome
Title Percent of Participants Who Achieved Improvement in Motor Milestones as Assessed by Section 2 of the HINE at the Last Visit
Hide Description Section 2 of HINE consists of 8 independent milestone categories. Within each of these categories, participants can progress from complete absence of a motor ability (the lowest level in each category) through multiple milestones (2 to 4 levels in each category) to the highest level within the category. Overall, there are a total of 26 milestones that can be achieved across the 8 categories. Improvement was defined as any of the following: 1. An increase from baseline of 2 milestones or more, or the achievement of pincer grasp in the voluntary grasp category 2. An increase from baseline of 2 milestones or more, or achievement of touching toes in the ability to kick category 3. An increase from baseline of 1 milestone or more in any of the remaining 6 categories: head control, rolling, sitting, crawling, standing, or walking.
Time Frame Day 1352 or Early Termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who were registered and received at least 1 dose of nusinersen.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Measure Type: Number
Unit of Measure: Percent of participants
25.0 68.8
2.Secondary Outcome
Title Event-free Survival at the End of Study
Hide Description Event-free survival was defined as the percent of participants who were alive and did not require permanent ventilatory support (defined as tracheostomy or the need for ≥16 hours ventilation/day continuously for at least 2 weeks in the absence of an acute reversible illness) Event-free survival was estimated using Kaplan-Meier methodology.
Time Frame Up to Day 1638
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who were registered and received at least 1 dose of nusinersen.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Measure Type: Number
Unit of Measure: Percent of participants
25.0 62.5
3.Secondary Outcome
Title Percent of Participants With Improved Motor Function at the Last Visit as Assessed by the CHOP-INTEND Motor Function Scale
Hide Description The CHOP-INTEND test includes 16 items structured to move from easiest to hardest with the grading including gravity eliminated (lower scores) to antigravity movements (higher scores). All item scores range from 0 (worst) to 4 (best). Total scores range from 0 to 64, with higher scores indicating better movement functioning. Improvement was defined as an increase in total CHOP INTEND score ≥4 points from baseline as of the last study visit.
Time Frame Day 1352 or Early Termination
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who were registered and received at least 1 dose of nusinersen.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Measure Type: Number
Unit of Measure: Percent of participants
25.0 62.5
4.Secondary Outcome
Title Change in Neuromuscular Electrophysiology at the Last Visit as Assessed by the Change From Baseline in CMAP Amplitude
Hide Description 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 positive change from Baseline indicates that the number of motor neurons increased.
Time Frame Baseline, Day 1072
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who were registered and received at least 1 dose of nusinersen. Here, number of participants analyzed is the participants who were evaluable for this outcome measure.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 2 8
Mean (Standard Deviation)
Unit of Measure: mV
Peroneal nerve 1.88  (2.58) 2.81  (1.28)
Ulnar nerve 0.776  (1.448) 0.685  (0.415)
5.Secondary Outcome
Title Number of Participants Experiencing Adverse Events (AEs) and/or Serious Adverse Events (SAEs)
Hide Description 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 subject (whether male or female); is an important medical event in the opinion of the Investigator or Sponsor.
Time Frame Up to Day 1352
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who were registered and received at least 1 dose of nusinersen.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Measure Type: Number
Unit of Measure: participants
Any AE 4 16
Any SAE 3 13
6.Secondary Outcome
Title Concentration of Nusinersen in Cerebrospinal Fluid (CSF)
Hide Description The concentration of nusinersen in CSF was measured by using standard laboratory assays.
Time Frame Day 1135 (Predose)
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population: All participants who were registered and had at least 1 evaluable postdose PK sample. Here, number of participants analyzed is the participants who were evaluable for this outcome measure.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 2 6
Mean (Standard Deviation)
Unit of Measure: nanograms/milliliter (ng/mL)
12.2  (8.60) 11.1  (4.99)
7.Secondary Outcome
Title PK Parameters of Nusinersen in Plasma: Maximum Concentration (Cmax)
Hide Description Cmax is the maximum observed concentration of study drug in plasma.
Time Frame Day 1 (Predose and 1, 2, and 4 hours [hr] Postdose) and Day 2 (24 hr Postdose )
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population: All participants who were registered and had at least 1 evaluable postdose PK sample.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Mean (Standard Deviation)
Unit of Measure: ng/mL
396  (311) 829  (625)
8.Secondary Outcome
Title PK Parameters of Nusinersen in Plasma: Time to Reach Cmax (Tmax)
Hide Description Tmax is the time at which Cmax occurs.
Time Frame Day 1 (Predose and 1, 2, and 4 hours [hr] Postdose) and Day 2 (24 hr Postdose )
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population: All participants who were registered and had at least 1 evaluable postdose PK sample.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Mean (Standard Deviation)
Unit of Measure: hr
2.09  (1.35) 2.37  (1.25)
9.Secondary Outcome
Title PK Parameters of Nusinersen in Plasma: Area Under the Plasma Concentrations Time Curve From the Time of the IT Dose to Four Hours After Dosing (AUC0-4)
Hide Description AUC is area under the plasma concentration-time curve from zero time (Predose) to 4 hours after IT administration of study drug. AUC was determined by using the linear trapezoidal rule.
Time Frame Day 1 (Predose and 1, 2, and 4 hours [hr] Postdose) and Day 2 (24 hr Postdose )
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population: All participants who were registered and had at least 1 evaluable postdose PK sample.
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description:
Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Participants received nusinersen 12 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261.
Overall Number of Participants Analyzed 4 16
Mean (Standard Deviation)
Unit of Measure: ng x hr/mL
894  (610) 2181  (1488)
Time Frame Up to Day 1359
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Nusinersen 6 mg Nusinersen 12 mg
Hide Arm/Group Description Participants received nusinersen 6 mg injections as an intrathecal (IT) bolus through a lumbar puncture (LP) on Days 1, 15, and 85. Maintenance doses of 12 mg were given on Days 253, 379, 505, 631, 757, 883, 1009, 1135, and 1261. Participants aged 21 days to 7 months old with infantile-onset SMA received nusinersen 12 mg injections at regular intervals for up to 45 months.
All-Cause Mortality
Nusinersen 6 mg Nusinersen 12 mg
Affected / at Risk (%) Affected / at Risk (%)
Total   1/4 (25.00%)   4/16 (25.00%) 
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Nusinersen 6 mg Nusinersen 12 mg
Affected / at Risk (%) Affected / at Risk (%)
Total   3/4 (75.00%)   13/16 (81.25%) 
Cardiac disorders     
Bradycardia  1  0/4 (0.00%)  1/16 (6.25%) 
Cardiac arrest  1  0/4 (0.00%)  1/16 (6.25%) 
Cardio-respiratory arrest  1  0/4 (0.00%)  1/16 (6.25%) 
Cyanosis  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumopericardium  1  0/4 (0.00%)  1/16 (6.25%) 
Gastrointestinal disorders     
Vomiting  1  0/4 (0.00%)  1/16 (6.25%) 
Infections and infestations     
Adenovirus infection  1  0/4 (0.00%)  1/16 (6.25%) 
Bronchiolitis  1  0/4 (0.00%)  3/16 (18.75%) 
Corona virus infection  1  1/4 (25.00%)  1/16 (6.25%) 
Enterovirus infection  1  0/4 (0.00%)  1/16 (6.25%) 
Lower respiratory tract infection viral  1  0/4 (0.00%)  1/16 (6.25%) 
Metapneumovirus infection  1  0/4 (0.00%)  2/16 (12.50%) 
Parainfluenzae virus infection  1  1/4 (25.00%)  0/16 (0.00%) 
Pneumonia  1  0/4 (0.00%)  5/16 (31.25%) 
Pneumonia adenoviral  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumonia bacterial  1  1/4 (25.00%)  1/16 (6.25%) 
Pneumonia pseudomonas aeruginosa  1  1/4 (25.00%)  1/16 (6.25%) 
Pneumonia viral  1  0/4 (0.00%)  3/16 (18.75%) 
Respiratory syncytial virus bronchiolitis  1  1/4 (25.00%)  3/16 (18.75%) 
Respiratory tract infection  1  1/4 (25.00%)  0/16 (0.00%) 
Respiratory tract infection viral  1  0/4 (0.00%)  1/16 (6.25%) 
Rhinovirus infection  1  0/4 (0.00%)  4/16 (25.00%) 
Upper respiratory tract infection  1  0/4 (0.00%)  1/16 (6.25%) 
Viral infection  1  0/4 (0.00%)  2/16 (12.50%) 
Viral upper respiratory tract infection  1  0/4 (0.00%)  2/16 (12.50%) 
Metabolism and nutrition disorders     
Failure to thrive  1  0/4 (0.00%)  1/16 (6.25%) 
Hyponatraemia  1  0/4 (0.00%)  1/16 (6.25%) 
Musculoskeletal and connective tissue disorders     
Synovitis  1  0/4 (0.00%)  1/16 (6.25%) 
Nervous system disorders     
Convulsion  1  0/4 (0.00%)  1/16 (6.25%) 
Hypoxic-ischaemic encephalopathy  1  0/4 (0.00%)  1/16 (6.25%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory failure  1  2/4 (50.00%)  4/16 (25.00%) 
Apnoea  1  0/4 (0.00%)  2/16 (12.50%) 
Asphyxia  1  1/4 (25.00%)  0/16 (0.00%) 
Aspiration  1  0/4 (0.00%)  1/16 (6.25%) 
Atelectasis  1  0/4 (0.00%)  2/16 (12.50%) 
Bronchial secretion retention  1  0/4 (0.00%)  1/16 (6.25%) 
Chronic respiratory failure  1  1/4 (25.00%)  0/16 (0.00%) 
Hypoventilation  1  0/4 (0.00%)  1/16 (6.25%) 
Hypoxia  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumomediastinum  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumonia aspiration  1  0/4 (0.00%)  3/16 (18.75%) 
Respiratory distress  1  2/4 (50.00%)  6/16 (37.50%) 
Respiratory failure  1  0/4 (0.00%)  5/16 (31.25%) 
1
Term from vocabulary, MedDRA 16.0
Indicates events were collected by systematic assessment
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Frequency Threshold for Reporting Other Adverse Events 0%
Nusinersen 6 mg Nusinersen 12 mg
Affected / at Risk (%) Affected / at Risk (%)
Total   4/4 (100.00%)   16/16 (100.00%) 
Blood and lymphatic system disorders     
Anaemia  1  0/4 (0.00%)  2/16 (12.50%) 
Eosinophilia  1  0/4 (0.00%)  1/16 (6.25%) 
Lymphadenopathy  1  1/4 (25.00%)  1/16 (6.25%) 
Neutropenia  1  0/4 (0.00%)  1/16 (6.25%) 
Thrombocytosis  1  0/4 (0.00%)  1/16 (6.25%) 
Cardiac disorders     
Bradycardia  1  1/4 (25.00%)  1/16 (6.25%) 
Cardiomyopathy  1  0/4 (0.00%)  1/16 (6.25%) 
Tachycardia  1  1/4 (25.00%)  2/16 (12.50%) 
Ventricular tachycardia  1  1/4 (25.00%)  0/16 (0.00%) 
Congenital, familial and genetic disorders     
Clinodactyly  1  0/4 (0.00%)  1/16 (6.25%) 
Cryptorchism  1  1/4 (25.00%)  1/16 (6.25%) 
High arched palate  1  1/4 (25.00%)  0/16 (0.00%) 
Hip dysplasia  1  0/4 (0.00%)  1/16 (6.25%) 
Pectus carinatum  1  0/4 (0.00%)  1/16 (6.25%) 
Plagiocephaly  1  1/4 (25.00%)  1/16 (6.25%) 
Ear and labyrinth disorders     
Cerumen impaction  1  1/4 (25.00%)  1/16 (6.25%) 
Middle ear effusion  1  0/4 (0.00%)  1/16 (6.25%) 
Eye disorders     
Astigmatism  1  1/4 (25.00%)  0/16 (0.00%) 
Conjunctivitis  1  0/4 (0.00%)  1/16 (6.25%) 
Erythema of eyelid  1  0/4 (0.00%)  1/16 (6.25%) 
Eye irritation  1  0/4 (0.00%)  1/16 (6.25%) 
Eyelid ptosis  1  0/4 (0.00%)  1/16 (6.25%) 
Ocular hyperaemia  1  0/4 (0.00%)  1/16 (6.25%) 
Gastrointestinal disorders     
Abdominal distension  1  1/4 (25.00%)  2/16 (12.50%) 
Abdominal pain  1  1/4 (25.00%)  0/16 (0.00%) 
Chapped lips  1  0/4 (0.00%)  2/16 (12.50%) 
Constipation  1  1/4 (25.00%)  8/16 (50.00%) 
Diarrhoea  1  2/4 (50.00%)  4/16 (25.00%) 
Flatulence  1  1/4 (25.00%)  1/16 (6.25%) 
Frequent bowel movements  1  0/4 (0.00%)  1/16 (6.25%) 
Gastritis  1  0/4 (0.00%)  1/16 (6.25%) 
Gastrointestinal hypomotility  1  1/4 (25.00%)  0/16 (0.00%) 
Gastrooesophageal reflux disease  1  0/4 (0.00%)  6/16 (37.50%) 
Haematemesis  1  1/4 (25.00%)  0/16 (0.00%) 
Salivary hypersecretion  1  0/4 (0.00%)  2/16 (12.50%) 
Stomatitis  1  0/4 (0.00%)  1/16 (6.25%) 
Teething  1  2/4 (50.00%)  2/16 (12.50%) 
Tongue atrophy  1  0/4 (0.00%)  2/16 (12.50%) 
Tooth crowding  1  1/4 (25.00%)  0/16 (0.00%) 
Traumatic tooth displacement  1  0/4 (0.00%)  1/16 (6.25%) 
Vomiting  1  2/4 (50.00%)  5/16 (31.25%) 
General disorders     
Cyst  1  0/4 (0.00%)  1/16 (6.25%) 
Device leakage  1  0/4 (0.00%)  1/16 (6.25%) 
Device occlusion  1  0/4 (0.00%)  1/16 (6.25%) 
Discomfort  1  1/4 (25.00%)  1/16 (6.25%) 
Infusion site extravasation  1  0/4 (0.00%)  1/16 (6.25%) 
Injection site bruising  1  0/4 (0.00%)  1/16 (6.25%) 
Non-cardiac chest pain  1  0/4 (0.00%)  1/16 (6.25%) 
Oedema  1  0/4 (0.00%)  2/16 (12.50%) 
Oedema peripheral  1  2/4 (50.00%)  3/16 (18.75%) 
Pain  1  0/4 (0.00%)  4/16 (25.00%) 
Pyrexia  1  3/4 (75.00%)  14/16 (87.50%) 
Vessel puncture site bruise  1  0/4 (0.00%)  1/16 (6.25%) 
Vessel puncture site haemorrhage  1  0/4 (0.00%)  1/16 (6.25%) 
Immune system disorders     
Drug hypersensitivity  1  0/4 (0.00%)  2/16 (12.50%) 
Hypersensitivity  1  0/4 (0.00%)  1/16 (6.25%) 
Seasonal allergy  1  1/4 (25.00%)  3/16 (18.75%) 
Infections and infestations     
Adenoviral upper respiratory infection  1  0/4 (0.00%)  1/16 (6.25%) 
Adenovirus infection  1  0/4 (0.00%)  1/16 (6.25%) 
Bacterial disease carrier  1  0/4 (0.00%)  1/16 (6.25%) 
Bacterial infection  1  0/4 (0.00%)  1/16 (6.25%) 
Bacterial tracheitis  1  0/4 (0.00%)  2/16 (12.50%) 
Bronchiolitis  1  0/4 (0.00%)  1/16 (6.25%) 
Candida nappy rash  1  1/4 (25.00%)  0/16 (0.00%) 
Ear infection  1  0/4 (0.00%)  3/16 (18.75%) 
Enterovirus infection  1  0/4 (0.00%)  2/16 (12.50%) 
Erythema infectiosum  1  0/4 (0.00%)  1/16 (6.25%) 
Fungal skin infection  1  0/4 (0.00%)  1/16 (6.25%) 
Gastritis viral  1  0/4 (0.00%)  1/16 (6.25%) 
Gastroenteritis  1  0/4 (0.00%)  1/16 (6.25%) 
Gastroenteritis viral  1  0/4 (0.00%)  1/16 (6.25%) 
Haemophilus infection  1  0/4 (0.00%)  1/16 (6.25%) 
Hand-foot-and-mouth disease  1  0/4 (0.00%)  1/16 (6.25%) 
Hordeolum  1  0/4 (0.00%)  1/16 (6.25%) 
Influenza  1  0/4 (0.00%)  1/16 (6.25%) 
Lower respiratory tract infection  1  0/4 (0.00%)  1/16 (6.25%) 
Moraxella infection  1  0/4 (0.00%)  1/16 (6.25%) 
Mucocutaneous candidiasis  1  0/4 (0.00%)  1/16 (6.25%) 
Nasopharyngitis  1  1/4 (25.00%)  7/16 (43.75%) 
Oral candidiasis  1  0/4 (0.00%)  1/16 (6.25%) 
Otitis media  1  1/4 (25.00%)  6/16 (37.50%) 
Parainfluenzae virus infection  1  1/4 (25.00%)  1/16 (6.25%) 
Pharyngitis streptococcal  1  1/4 (25.00%)  0/16 (0.00%) 
Pneumonia  1  1/4 (25.00%)  5/16 (31.25%) 
Pneumonia moraxella  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumonia pseudomonas aeruginosa  1  1/4 (25.00%)  1/16 (6.25%) 
Pneumonia streptococcal  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumonia viral  1  1/4 (25.00%)  0/16 (0.00%) 
Pseudomonas infection  1  0/4 (0.00%)  1/16 (6.25%) 
Respiratory syncytial virus bronchiolitis  1  1/4 (25.00%)  1/16 (6.25%) 
Respiratory syncytial virus infection  1  0/4 (0.00%)  1/16 (6.25%) 
Respiratory tract infection  1  3/4 (75.00%)  4/16 (25.00%) 
Respiratory tract infection bacterial  1  0/4 (0.00%)  1/16 (6.25%) 
Rhinitis  1  0/4 (0.00%)  3/16 (18.75%) 
Rhinovirus infection  1  0/4 (0.00%)  4/16 (25.00%) 
Staphylococcal bacteraemia  1  0/4 (0.00%)  1/16 (6.25%) 
Staphylococcal infection  1  0/4 (0.00%)  1/16 (6.25%) 
Tracheitis  1  0/4 (0.00%)  2/16 (12.50%) 
Upper respiratory tract infection  1  1/4 (25.00%)  10/16 (62.50%) 
Urinary tract infection  1  0/4 (0.00%)  1/16 (6.25%) 
Viral infection  1  0/4 (0.00%)  4/16 (25.00%) 
Viral rash  1  0/4 (0.00%)  1/16 (6.25%) 
Viral upper respiratory tract infection  1  0/4 (0.00%)  2/16 (12.50%) 
Injury, poisoning and procedural complications     
Arthropod bite  1  1/4 (25.00%)  2/16 (12.50%) 
Contusion  1  0/4 (0.00%)  1/16 (6.25%) 
Ear abrasion  1  0/4 (0.00%)  1/16 (6.25%) 
Excoriation  1  1/4 (25.00%)  2/16 (12.50%) 
Feeding tube complication  1  0/4 (0.00%)  3/16 (18.75%) 
Joint dislocation  1  1/4 (25.00%)  3/16 (18.75%) 
Laceration  1  0/4 (0.00%)  2/16 (12.50%) 
Lip injury  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumothorax traumatic  1  0/4 (0.00%)  1/16 (6.25%) 
Post gastric surgery syndrome  1  0/4 (0.00%)  1/16 (6.25%) 
Procedural pain  1  1/4 (25.00%)  1/16 (6.25%) 
Procedural site reaction  1  0/4 (0.00%)  1/16 (6.25%) 
Soft tissue injury  1  1/4 (25.00%)  0/16 (0.00%) 
Sunburn  1  1/4 (25.00%)  0/16 (0.00%) 
Thermal burn  1  1/4 (25.00%)  0/16 (0.00%) 
Tooth injury  1  0/4 (0.00%)  1/16 (6.25%) 
Urinary retention postoperative  1  1/4 (25.00%)  0/16 (0.00%) 
Investigations     
Bacterial test positive  1  1/4 (25.00%)  0/16 (0.00%) 
Bone density decreased  1  0/4 (0.00%)  2/16 (12.50%) 
Breath sounds abnormal  1  0/4 (0.00%)  1/16 (6.25%) 
Heart rate decreased  1  0/4 (0.00%)  1/16 (6.25%) 
Heart rate increased  1  0/4 (0.00%)  1/16 (6.25%) 
Human rhinovirus test positive  1  0/4 (0.00%)  3/16 (18.75%) 
Moraxella test positive  1  0/4 (0.00%)  1/16 (6.25%) 
Neutrophil count increased  1  0/4 (0.00%)  2/16 (12.50%) 
Neutrophil percentage increased  1  0/4 (0.00%)  1/16 (6.25%) 
Oxygen saturation decreased  1  0/4 (0.00%)  2/16 (12.50%) 
Pseudomonas test positive  1  0/4 (0.00%)  1/16 (6.25%) 
Serratia test positive  1  0/4 (0.00%)  1/16 (6.25%) 
Staphylococcus test positive  1  0/4 (0.00%)  1/16 (6.25%) 
Streptococcus test positive  1  0/4 (0.00%)  1/16 (6.25%) 
Weight decreased  1  1/4 (25.00%)  1/16 (6.25%) 
White blood cell count increased  1  0/4 (0.00%)  2/16 (12.50%) 
Metabolism and nutrition disorders     
Dehydration  1  1/4 (25.00%)  0/16 (0.00%) 
Failure to thrive  1  0/4 (0.00%)  1/16 (6.25%) 
Hyperglycaemia  1  0/4 (0.00%)  1/16 (6.25%) 
Hyperkalaemia  1  1/4 (25.00%)  0/16 (0.00%) 
Hyponatraemia  1  0/4 (0.00%)  2/16 (12.50%) 
Hypophagia  1  1/4 (25.00%)  1/16 (6.25%) 
Malnutrition  1  1/4 (25.00%)  0/16 (0.00%) 
Metabolic alkalosis  1  0/4 (0.00%)  1/16 (6.25%) 
Weight gain poor  1  1/4 (25.00%)  0/16 (0.00%) 
Musculoskeletal and connective tissue disorders     
Arthritis  1  0/4 (0.00%)  1/16 (6.25%) 
Deformity thorax  1  1/4 (25.00%)  2/16 (12.50%) 
Hand deformity  1  0/4 (0.00%)  1/16 (6.25%) 
Joint contracture  1  3/4 (75.00%)  7/16 (43.75%) 
Joint range of motion decreased  1  0/4 (0.00%)  1/16 (6.25%) 
Joint stiffness  1  0/4 (0.00%)  1/16 (6.25%) 
Kyphoscoliosis  1  0/4 (0.00%)  2/16 (12.50%) 
Kyphosis  1  2/4 (50.00%)  3/16 (18.75%) 
Lordosis  1  0/4 (0.00%)  1/16 (6.25%) 
Osteopenia  1  1/4 (25.00%)  0/16 (0.00%) 
Pain in extremity  1  0/4 (0.00%)  2/16 (12.50%) 
Rib deformity  1  0/4 (0.00%)  1/16 (6.25%) 
Scoliosis  1  2/4 (50.00%)  6/16 (37.50%) 
Spinal deformity  1  0/4 (0.00%)  1/16 (6.25%) 
Synovitis  1  0/4 (0.00%)  1/16 (6.25%) 
Torticollis  1  2/4 (50.00%)  2/16 (12.50%) 
Trismus  1  1/4 (25.00%)  0/16 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Acrochordon  1  0/4 (0.00%)  1/16 (6.25%) 
Haemangioma  1  1/4 (25.00%)  0/16 (0.00%) 
Nervous system disorders     
Cerebral infarction  1  0/4 (0.00%)  1/16 (6.25%) 
Cerebrospinal fluid leakage  1  0/4 (0.00%)  1/16 (6.25%) 
Convulsion  1  0/4 (0.00%)  1/16 (6.25%) 
Drooling  1  0/4 (0.00%)  1/16 (6.25%) 
Headache  1  1/4 (25.00%)  0/16 (0.00%) 
Hyporeflexia  1  0/4 (0.00%)  1/16 (6.25%) 
Lethargy  1  0/4 (0.00%)  1/16 (6.25%) 
Muscle contractions involuntary  1  1/4 (25.00%)  3/16 (18.75%) 
Nystagmus  1  1/4 (25.00%)  0/16 (0.00%) 
Speech disorder developmental  1  1/4 (25.00%)  2/16 (12.50%) 
Syncope  1  0/4 (0.00%)  1/16 (6.25%) 
Psychiatric disorders     
Anxiety  1  0/4 (0.00%)  3/16 (18.75%) 
Expressive language disorder  1  0/4 (0.00%)  1/16 (6.25%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory failure  1  1/4 (25.00%)  0/16 (0.00%) 
Apnoea  1  0/4 (0.00%)  2/16 (12.50%) 
Apnoeic attack  1  0/4 (0.00%)  1/16 (6.25%) 
Aspiration  1  0/4 (0.00%)  1/16 (6.25%) 
Atelectasis  1  0/4 (0.00%)  5/16 (31.25%) 
Bronchial secretion retention  1  1/4 (25.00%)  2/16 (12.50%) 
Choking  1  0/4 (0.00%)  1/16 (6.25%) 
Chronic respiratory failure  1  2/4 (50.00%)  3/16 (18.75%) 
Cough  1  2/4 (50.00%)  5/16 (31.25%) 
Dyspnoea  1  1/4 (25.00%)  1/16 (6.25%) 
Epistaxis  1  0/4 (0.00%)  1/16 (6.25%) 
Hypoxia  1  0/4 (0.00%)  4/16 (25.00%) 
Increased bronchial secretion  1  0/4 (0.00%)  1/16 (6.25%) 
Increased upper airway secretion  1  0/4 (0.00%)  5/16 (31.25%) 
Laryngeal granuloma  1  0/4 (0.00%)  1/16 (6.25%) 
Nasal congestion  1  1/4 (25.00%)  6/16 (37.50%) 
Nasal discharge discolouration  1  0/4 (0.00%)  1/16 (6.25%) 
Pneumonia aspiration  1  0/4 (0.00%)  2/16 (12.50%) 
Pneumothorax  1  0/4 (0.00%)  2/16 (12.50%) 
Pulmonary congestion  1  0/4 (0.00%)  2/16 (12.50%) 
Pulmonary oedema  1  0/4 (0.00%)  2/16 (12.50%) 
Respiratory arrest  1  0/4 (0.00%)  1/16 (6.25%) 
Respiratory distress  1  0/4 (0.00%)  3/16 (18.75%) 
Respiratory failure  1  0/4 (0.00%)  2/16 (12.50%) 
Restrictive pulmonary disease  1  0/4 (0.00%)  1/16 (6.25%) 
Rhinitis allergic  1  1/4 (25.00%)  0/16 (0.00%) 
Rhinorrhoea  1  0/4 (0.00%)  2/16 (12.50%) 
Rhonchi  1  0/4 (0.00%)  1/16 (6.25%) 
Sinus congestion  1  0/4 (0.00%)  1/16 (6.25%) 
Sleep apnoea syndrome  1  0/4 (0.00%)  1/16 (6.25%) 
Tachypnoea  1  0/4 (0.00%)  1/16 (6.25%) 
Tracheal disorder  1  0/4 (0.00%)  1/16 (6.25%) 
Upper respiratory tract congestion  1  0/4 (0.00%)  1/16 (6.25%) 
Use of accessory respiratory muscles  1  0/4 (0.00%)  1/16 (6.25%) 
Wheezing  1  0/4 (0.00%)  2/16 (12.50%) 
Skin and subcutaneous tissue disorders     
Alopecia  1  0/4 (0.00%)  1/16 (6.25%) 
Blister  1  1/4 (25.00%)  0/16 (0.00%) 
Dermatitis  1  0/4 (0.00%)  1/16 (6.25%) 
Dermatitis contact  1  1/4 (25.00%)  0/16 (0.00%) 
Dermatitis diaper  1  2/4 (50.00%)  3/16 (18.75%) 
Ecchymosis  1  1/4 (25.00%)  2/16 (12.50%) 
Eczema  1  1/4 (25.00%)  3/16 (18.75%) 
Erythema  1  1/4 (25.00%)  1/16 (6.25%) 
Excessive granulation tissue  1  0/4 (0.00%)  3/16 (18.75%) 
Heat rash  1  1/4 (25.00%)  0/16 (0.00%) 
Petechiae  1  0/4 (0.00%)  1/16 (6.25%) 
Pruritus  1  0/4 (0.00%)  1/16 (6.25%) 
Rash  1  1/4 (25.00%)  5/16 (31.25%) 
Rash follicular  1  0/4 (0.00%)  1/16 (6.25%) 
Rash papular  1  0/4 (0.00%)  1/16 (6.25%) 
Rosacea  1  0/4 (0.00%)  1/16 (6.25%) 
Skin depigmentation  1  0/4 (0.00%)  1/16 (6.25%) 
Skin disorder  1  1/4 (25.00%)  0/16 (0.00%) 
Skin irritation  1  1/4 (25.00%)  3/16 (18.75%) 
Skin ulcer  1  0/4 (0.00%)  1/16 (6.25%) 
Subcutaneous emphysema  1  0/4 (0.00%)  1/16 (6.25%) 
Vascular disorders     
Capillary fragility  1  0/4 (0.00%)  1/16 (6.25%) 
Essential hypertension  1  1/4 (25.00%)  0/16 (0.00%) 
Hypertension  1  0/4 (0.00%)  1/16 (6.25%) 
Subgaleal haematoma  1  0/4 (0.00%)  1/16 (6.25%) 
1
Term from vocabulary, MedDRA 16.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: US Biogen Clinical Trial Center
Organization: Biogen
Phone: 866-633-4636
EMail: clinicaltrials@biogen.com
Layout table for additonal information
Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT01839656     History of Changes
Other Study ID Numbers: ISIS 396443-CS3A
2017-000621-12 ( EudraCT Number )
First Submitted: April 22, 2013
First Posted: April 25, 2013
Results First Submitted: August 16, 2018
Results First Posted: October 30, 2018
Last Update Posted: October 30, 2018