| Trial name or title |
A phase II, randomised, double‐blind, sham‐procedure controlled study to assess the safety and tolerability and explore the efficacy of IONIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies IONIS 396443‐CS3B or IONIS 396443‐CS4 |
| Methods |
Phase II, randomised, double‐blind, sham‐procedure‐controlled study |
| Participants |
21 participants with genetically confirmed SMA with onset of clinical signs and symptoms consistent with SMA at ≤ 6 months of age and have documentation of 3 SMN2 copies OR onset of clinical signs and symptoms consistent with SMA at ≤ 6 months of age, > 7 months of age (211 days) at screening, and have documentation of 2 SMN2 copies OR onset of clinical signs and symptoms consistent with SMA at > 6 months of age, are ≤ 18 months of age at screening, and have documentation of 2 or 3 SMN2 copies |
| Interventions |
Multiple intrathecal injection of nusinersen (also called IONIS‐SMN Rx or IONIS 396443) or multiple sham procedure with placebo |
| Outcomes |
Number of adverse events and serious adverse events, change from baseline in clinical laboratory parameters, change from baseline in electrocardiogram, change from baseline in vital signs, change from baseline in neurological examination including motor function, change in plasma concentration of nusinersen and change in cerebrospinal fluid concentration of nusinersen |
| Starting date |
June 2015 |
| Contact information |
Biogen |
| Notes |
Study ongoing, but not recruiting participants. |
