The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS1 (NCT02865109). The secondary objective was to examine the plasma pharmacokinetics of a single dose of ISIS 396443 administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS1.
This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, sponsorship of the trial was transferred to Biogen.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
18
Administered by intrathecal (IT) injection
Boston Children's Hospital
Boston, Massachusetts, United States
Columbia University Medical Center
New York, New York, United States
UT Southwestern Medical Center - Children's Medical Center Dallas
Dallas, Texas, United States
University of Utah School of Medicine
Salt Lake City, Utah, United States
Number of participants that experience Adverse Events (AEs) and Serious Adverse Events
Time frame: Up to 24 Weeks
Number of participants with clinically significant neurological examination abnormalities
Time frame: Up tp 24 Weeks
Number of participants with clinically significant vital sign abnormalities
Time frame: Up to 24 Weeks
Number of participants with clinically significant physical examination abnormalities
Time frame: Up to 24 Weeks
Number of participants with clinically significant weight abnormalities
Time frame: Up to 24 Weeks
Number of participants with clinically significant laboratory parameters
Time frame: Up to 24 Weeks
Number of participants with clinically significant electrocardiograms (ECGs) abnormalities
Time frame: Up to 24 Weeks
Number of participants who use concomitant medications
Time frame: Up to 24 Weeks
PK parameters of nusinersen (ISIS 396443): Maximum observed plasma drug concentration (Cmax)
Time frame: Plasma at 1, 2, 4 and 6 hours after dosing
PK parameters of nusinersen: Time to reach maximum observed concentration (Tmax)
Time frame: Plasma at 1, 2, 4 and 6 hours after dosing
PK parameters of nusinersen: Area under the plasma concentrations time curve from the time of the intrathecal (IT) dose to the last collected sample (AUCinf)
Time frame: Plasma at 1, 2, 4 and 6 hours after dosing
PK parameters of nusinersen (ISIS 396443): Apparent terminal elimination half-life (t1/2), if possible
Time frame: Plasma at 1, 2, 4 and 6 hours after dosing
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