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An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01703988
Recruitment Status : Completed
First Posted : October 11, 2012
Results First Posted : March 13, 2017
Last Update Posted : March 13, 2017
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 34
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description 3 mg nusinersen on Days 1, 29, 85, intrathecal (IT) injection 6 mg nusinersen on Days 1, 29, 85, IT injection 9 mg nusinersen on Days 1 and 85, IT injection 12 mg nusinersen on Days 1, 29, 85, IT injection
Period Title: Overall Study
Started 8 8 9 9
Completed 8 8 9 8
Not Completed 0 0 0 1
Reason Not Completed
Investigator Judgment             0             0             0             1
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg Total
Hide Arm/Group Description 3 mg nusinersen on Days 1, 29, 85, IT injection 6 mg nusinersen on Days 1, 29, 85, IT injection 9 mg nusinersen on Days 1 and 85, IT injection 12 mg nusinersen on Days 1, 29, 85, IT injection Total of all reporting groups
Overall Number of Baseline Participants 8 8 9 9 34
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 8 participants 9 participants 9 participants 34 participants
8.6  (5.4) 8.1  (5.2) 6.0  (4.0) 6.9  (4.3) 7.4  (4.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 8 participants 9 participants 9 participants 34 participants
Female
4
  50.0%
3
  37.5%
4
  44.4%
3
  33.3%
14
  41.2%
Male
4
  50.0%
5
  62.5%
5
  55.6%
6
  66.7%
20
  58.8%
1.Primary Outcome
Title Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs
Hide Description An AE is any unfavorable and unintended sign, symptom, or disease temporally associated with the study or use of the investigational drug product, whether or not the AE is considered related to the investigational drug product. An SAE is any AE that, in the view of either the Investigator or Sponsor, meets any of the following criteria: results in death; is life threatening; requires inpatient 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; and is an important medical event in the judgment of the investigator. Drug-related is an event related or possibly related to study drug. Severity of AEs was assessed as mild, moderate, or severe.
Time Frame Participants were followed for the duration of the study; mean (SD) duration of treatment was 82.9 (15.4) days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population, defined as all enrolled participants who received at least 1 dose of study drug.
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description:
3 mg nusinersen on Days 1, 29, 85, IT injection
6 mg nusinersen on Days 1, 29, 85, IT injection
9 mg nusinersen on Days 1 and 85, IT injection
12 mg nusinersen on Days 1, 29, 85, IT injection
Overall Number of Participants Analyzed 8 8 9 9
Measure Type: Count of Participants
Unit of Measure: Participants
Any AE
8
 100.0%
6
  75.0%
9
 100.0%
9
 100.0%
Any drug-related AE
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Any SAE
2
  25.0%
0
   0.0%
0
   0.0%
1
  11.1%
Discontinued due to an AE
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Highest severity of AE=mild
2
  25.0%
2
  25.0%
6
  66.7%
5
  55.6%
Highest severity of AE=moderate
4
  50.0%
2
  25.0%
3
  33.3%
4
  44.4%
Highest severity of AE=severe
2
  25.0%
2
  25.0%
0
   0.0%
0
   0.0%
2.Secondary Outcome
Title Plasma Pharmacokinetics: Maximal Observed Plasma Drug Concentration (Cmax)
Hide Description [Not Specified]
Time Frame Day 1 and Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point.
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description:
3 mg nusinersen on Days 1, 29, 85, IT injection
6 mg nusinersen on Days 1, 29, 85, IT injection
9 mg nusinersen on Days 1 and 85, IT injection
12 mg nusinersen on Days 1, 29, 85, IT injection
Overall Number of Participants Analyzed 8 8 9 9
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 8 participants 8 participants 9 participants 9 participants
51.5  (72.7) 79.8  (57.2) 141.0  (52.8) 208.0  (110.0)
Day 85 Number Analyzed 8 participants 8 participants 9 participants 8 participants
32.5  (32.3) 52.8  (33.5) 127.0  (37.7) 132.0  (85.6)
3.Secondary Outcome
Title Plasma Pharmacokinetics: Time to Reach Cmax in Plasma
Hide Description [Not Specified]
Time Frame Day 1 and Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point.
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description:
3 mg nusinersen on Days 1, 29, 85, IT injection
6 mg nusinersen on Days 1, 29, 85, IT injection
9 mg nusinersen on Days 1 and 85, IT injection
12 mg nusinersen on Days 1, 29, 85, IT injection
Overall Number of Participants Analyzed 8 8 9 9
Median (Full Range)
Unit of Measure: hours
Day 1 Number Analyzed 8 participants 8 participants 9 participants 9 participants
5.09
(2.03 to 12.0)
5.93
(2.00 to 23.0)
3.92
(2.00 to 8.03)
4.05
(1.97 to 12.0)
Day 85 Number Analyzed 8 participants 8 participants 9 participants 8 participants
6.08
(4.12 to 6.17)
6.04
(5.97 to 6.25)
4.12
(2.00 to 6.25)
5.92
(1.95 to 6.07)
4.Secondary Outcome
Title Plasma Pharmacokinetics: Plasma Pharmacokinetics: Area Under the Plasma Concentration Time Curve From the Time of the IT Dose to 6 Hours After Dosing (AUC0-6hr)
Hide Description [Not Specified]
Time Frame Day 1 and Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point.
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description:
3 mg nusinersen on Days 1, 29, 85, IT injection
6 mg nusinersen on Days 1, 29, 85, IT injection
9 mg nusinersen on Days 1 and 85, IT injection
12 mg nusinersen on Days 1, 29, 85, IT injection
Overall Number of Participants Analyzed 8 8 9 9
Mean (Standard Deviation)
Unit of Measure: ng*hr/mL
Day 1 Number Analyzed 8 participants 8 participants 9 participants 9 participants
181  (225) 306  (259) 601  (249) 823  (442)
Day 85 Number Analyzed 8 participants 8 participants 9 participants 8 participants
110  (107) 179  (135) 524  (185) 555  (398)
5.Secondary Outcome
Title Cerebrospinal Fluid (CSF) Pharmacokinetics: Predose CSF Drug Concentrations
Hide Description [Not Specified]
Time Frame Day 1, Day 29, and Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
PK Population, defined as all enrolled participants who have evaluable PK data; number analyzed=participants with an assessment at given time point. (This study includes participants previously enrolled in Study ISIS 396443 - CS1 (NCT01494701).
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description:
3 mg nusinersen on Days 1, 29, 85, IT injection
6 mg nusinersen on Days 1, 29, 85, IT injection
9 mg nusinersen on Days 1 and 85, IT injection
12 mg nusinersen on Days 1, 29, 85, IT injection
Overall Number of Participants Analyzed 8 8 9 9
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 6 participants 8 participants 6 participants 9 participants
0.0651  (0.117) 0.0560  (0.104) NA [1]   (NA) NA [1]   (NA)
Day 29 Number Analyzed 7 participants 8 participants 0 participants 7 participants
1.41  (0.456) 2.65  (1.44) 2.22  (0.924)
Day 85 Number Analyzed 8 participants 8 participants 9 participants 7 participants
2.12  (0.573) 3.76  (2.05) 1.50  (0.447) 3.36  (1.04)
[1]
below the limit of quantification (50 pg/mL)
6.Secondary Outcome
Title Urine Pharmacokinetics: Renal Clearance, Cohort 4
Hide Description Renal clearance of nusinersen for participants was assessed in the 12 mg reporting group only, per protocol.
Time Frame Day 1 and Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) Population, defined as all enrolled participants who have evaluable PK data
Arm/Group Title Nusinersen 12 mg
Hide Arm/Group Description:
12 mg nusinersen on Days 1, 29, 85, IT injection
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mL/hr
Day 1 0.674  (0.602)
Day 85 77.5  (92.5)
Time Frame Participants were followed for the duration of the study; mean (SD) duration of treatment was 82.9 (15.4) days.
Adverse Event Reporting Description Treatment emergent AEs are presented regardless of seriousness or relationship to investigational product.
 
Arm/Group Title Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Hide Arm/Group Description 3 mg nusinersen on Days 1, 29, 85, IT injection 6 mg nusinersen on Days 1, 29, 85, IT injection 9 mg nusinersen on Days 1 and 85, IT injection 12 mg nusinersen on Days 1, 29, 85, IT injection
All-Cause Mortality
Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Hide Serious Adverse Events
Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/8 (25.00%)   0/8 (0.00%)   0/9 (0.00%)   1/9 (11.11%) 
Immune system disorders         
Drug hypersensitivity † 1 [1]  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Infections and infestations         
Pneumonia † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Injury, poisoning and procedural complications         
Post lumbar puncture syndrome † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.1
[1]
Event assessed as unlikely to be related to study drug.
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Nusinersen 3 mg Nusinersen 6 mg Nusinersen 9 mg Nusinersen 12 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   8/8 (100.00%)   6/8 (75.00%)   9/9 (100.00%)   9/9 (100.00%) 
Cardiac disorders         
Palpitations † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Ear and labyrinth disorders         
Ear pain † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Eye disorders         
Conjunctivitis † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Strabismus † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Gastrointestinal disorders         
Vomiting † 1  1/8 (12.50%)  1/8 (12.50%)  2/9 (22.22%)  2/9 (22.22%) 
Constipation † 1  0/8 (0.00%)  0/8 (0.00%)  2/9 (22.22%)  2/9 (22.22%) 
Nausea † 1  2/8 (25.00%)  0/8 (0.00%)  2/9 (22.22%)  0/9 (0.00%) 
Abdominal discomfort † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Diarrhoea † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Food poisoning † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Hypoaesthesia oral † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Lip pain † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Oral pain † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
General disorders         
Puncture site pain † 1  0/8 (0.00%)  3/8 (37.50%)  3/9 (33.33%)  2/9 (22.22%) 
Pyrexia † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  3/9 (33.33%) 
Fatigue † 1  1/8 (12.50%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Catheter site haemorrhage † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Catheter site pain † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Catheter site related reaction † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Chest discomfort † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Chills † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Feeling cold † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Gravitational oedema † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Oedema peripheral † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Pain † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Vessel puncture site haematoma † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Immune system disorders         
Hypersensitivity † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Seasonal allergy † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Infections and infestations         
Nasopharyngitis † 1  0/8 (0.00%)  1/8 (12.50%)  4/9 (44.44%)  3/9 (33.33%) 
Upper respiratory tract infection † 1  2/8 (25.00%)  1/8 (12.50%)  2/9 (22.22%)  1/9 (11.11%) 
Pharyngitis streptococcal † 1  3/8 (37.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Viral upper respiratory tract infection † 1  0/8 (0.00%)  1/8 (12.50%)  2/9 (22.22%)  0/9 (0.00%) 
Localised infection † 1  0/8 (0.00%)  0/8 (0.00%)  2/9 (22.22%)  0/9 (0.00%) 
Otitis media † 1  2/8 (25.00%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Pneumonia † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Sinusitis † 1  0/8 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  0/9 (0.00%) 
Viral infection † 1  0/8 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  0/9 (0.00%) 
Ear infection † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Eye infection staphylococcal † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Gastroenteritis viral † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Gastrointestinal viral infection † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Impetigo † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Influenza † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Respiratory syncytial virus infection † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Rhinitis † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Skin bacterial infection † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Urinary tract infection † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Injury, poisoning and procedural complications         
Post lumbar puncture syndrome † 1  3/8 (37.50%)  1/8 (12.50%)  3/9 (33.33%)  5/9 (55.56%) 
Arthropod bite † 1  0/8 (0.00%)  1/8 (12.50%)  2/9 (22.22%)  0/9 (0.00%) 
Laceration † 1  1/8 (12.50%)  0/8 (0.00%)  1/9 (11.11%)  1/9 (11.11%) 
Procedural pain † 1  1/8 (12.50%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Sunburn † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Torus fracture † 1  0/8 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  0/9 (0.00%) 
Agitation postoperative † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Femur fracture † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Limb injury † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Postoperative ileus † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Thermal burn † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Tibia fracture † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Investigations         
Blood bicarbonate decreased † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Weight decreased † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Metabolism and nutrition disorders         
Dehydration † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Musculoskeletal and connective tissue disorders         
Back pain † 1  1/8 (12.50%)  2/8 (25.00%)  2/9 (22.22%)  4/9 (44.44%) 
Joint contracture † 1  0/8 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  1/9 (11.11%) 
Scoliosis † 1  0/8 (0.00%)  1/8 (12.50%)  1/9 (11.11%)  1/9 (11.11%) 
Arthralgia † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  1/9 (11.11%) 
Myalgia † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Neck pain † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Pain in extremity † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Pelvic deformity † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Skin papilloma † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Nervous system disorders         
Headache † 1  4/8 (50.00%)  2/8 (25.00%)  1/9 (11.11%)  0/9 (0.00%) 
Dysgeusia † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Dyslexia † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Hypoaesthesia † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Paraesthesia † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Psychiatric disorders         
Agitation † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Attention deficit/hyperactivity disorder † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Dysphoria † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Parasomnia † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Respiratory, thoracic and mediastinal disorders         
Oropharyngeal pain † 1  1/8 (12.50%)  0/8 (0.00%)  1/9 (11.11%)  1/9 (11.11%) 
Rhinorrhoea † 1  1/8 (12.50%)  0/8 (0.00%)  1/9 (11.11%)  1/9 (11.11%) 
Epistaxis † 1  1/8 (12.50%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
Atelectasis † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Cough † 1  0/8 (0.00%)  0/8 (0.00%)  0/9 (0.00%)  1/9 (11.11%) 
Nasal congestion † 1  1/8 (12.50%)  0/8 (0.00%)  0/9 (0.00%)  0/9 (0.00%) 
Restrictive pulmonary disease † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Wheezing † 1  0/8 (0.00%)  1/8 (12.50%)  0/9 (0.00%)  0/9 (0.00%) 
Skin and subcutaneous tissue disorders         
Night sweats † 1  0/8 (0.00%)  0/8 (0.00%)  1/9 (11.11%)  0/9 (0.00%) 
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.1
[Not Specified]
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
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Name/Title: Biogen Study Medical Director
Organization: Biogen
EMail: clinicaltrials@biogen.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT01703988    
Other Study ID Numbers: ISIS 396443 - CS2
2017-000327-27 ( EudraCT Number )
First Submitted: October 8, 2012
First Posted: October 11, 2012
Results First Submitted: January 20, 2017
Results First Posted: March 13, 2017
Last Update Posted: March 13, 2017