A Phase 1 interventional study of nusinersen in Spinal Muscular Atrophy, sponsored by Biogen. Completed at 4 sites in United States. Open to participants aged 2 Years to 15 Years. Per ClinicalTrials.gov, last updated 2021-02-16.
Sponsored by Biogen · Phase 1, Interventional, and Treatment
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.
494 studies on the registry are indexed under Muscular Atrophy; 94 are open to participants now.
This study's enrollment of 18 is below the median of 33 across 335 interventional studies indexed under Muscular Atrophy.
Browse Muscular Atrophy studies →Biogen is the lead sponsor of 494 studies on the registry; 22 are open to participants now.
Of its 98 completed or terminated interventional studies of FDA-regulated products, 49 (50%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Drug: nusinersen
Administered by intrathecal (IT) injection
Also known as: Sprinraza, ISIS 396443, IONIS-SMN Rx, BIIB058
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
This study is completed, as verified in Feb 2021. You cannot join it, but the record below documents what was studied.
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