439 Studies Now Enrolling
An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy (SMA)
A study for infants with pre-symptomatic Spinal Muscular Atrophy (SMA)
| Sponsor: | Biogen |
| Enrolling: | Male and Female Patients |
| IRB Number: | AAAO9052 |
| U.S. Govt. ID: | NCT02386553 |
| Contact: | Rosangel Cruz: 212-305-1336 / rc2836@columbia.edu |
Additional Study Information:
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can prevent or delay the onset of symptoms of SMA.
This study is closed
