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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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Biogen
ClinicalTrials.gov Identifier:
NCT01703988
First received: October 8, 2012
Last updated: January 20, 2017
Last verified: January 2017
Results First Received: January 20, 2017  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: No masking;   Primary Purpose: Treatment
Condition: Spinal Muscular Atrophy
Intervention: Drug: Nusinersen

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Nusinersen 3 mg 3 mg nusinersen on Days 1, 29, 85, intrathecal (IT) injection
Nusinersen 6 mg 6 mg nusinersen on Days 1, 29, 85, IT injection
Nusinersen 9 mg 9 mg nusinersen on Days 1 and 85, IT injection
Nusinersen 12 mg 12 mg nusinersen on Days 1, 29, 85, IT injection

Participant Flow:   Overall Study
    Nusinersen 3 mg   Nusinersen 6 mg   Nusinersen 9 mg   Nusinersen 12 mg
STARTED   8   8   9   9 
COMPLETED   8   8   9   8 
NOT COMPLETED   0   0   0   1 
Investigator Judgment                0                0                0                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Nusinersen 3 mg 3 mg nusinersen on Days 1, 29, 85, IT injection
Nusinersen 6 mg 6 mg nusinersen on Days 1, 29, 85, IT injection
Nusinersen 9 mg 9 mg nusinersen on Days 1 and 85, IT injection
Nusinersen 12 mg 12 mg nusinersen on Days 1, 29, 85, IT injection
Total Total of all reporting groups

Baseline Measures
   Nusinersen 3 mg   Nusinersen 6 mg   Nusinersen 9 mg   Nusinersen 12 mg   Total 
Overall Participants Analyzed 
[Units: Participants]
 8   8   9   9   34 
Age 
[Units: Years]
Mean (Standard Deviation)
 8.6  (5.4)   8.1  (5.2)   6.0  (4.0)   6.9  (4.3)   7.4  (4.6) 
Sex: Female, Male 
[Units: Participants]
Count of 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% 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Participants With Adverse Events (AEs), Serious AEs (SAEs), Discontinuations Due to AEs, and Highest Severity of AEs   [ Time Frame: Participants were followed for the duration of the study; mean (SD) duration of treatment was 82.9 (15.4) days ]

2.  Secondary:   Plasma Pharmacokinetics: Maximal Observed Plasma Drug Concentration (Cmax)   [ Time Frame: Day 1 and Day 85 ]

3.  Secondary:   Plasma Pharmacokinetics: Time to Reach Cmax in Plasma   [ Time Frame: Day 1 and Day 85 ]

4.  Secondary:   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)   [ Time Frame: Day 1 and Day 85 ]

5.  Secondary:   Cerebrospinal Fluid (CSF) Pharmacokinetics: Predose CSF Drug Concentrations   [ Time Frame: Day 1, Day 29, and Day 85 ]

6.  Secondary:   Urine Pharmacokinetics: Renal Clearance, Cohort 4   [ Time Frame: Day 1 and Day 85 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Biogen Study Medical Director
Organization: Biogen
e-mail: clinicaltrials@biogen.com



Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT01703988     History of Changes
Other Study ID Numbers: ISIS 396443 - CS2
2017-000327-27 ( EudraCT Number )
Study First Received: October 8, 2012
Results First Received: January 20, 2017
Last Updated: January 20, 2017