A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Later-onset Spinal Muscular Atrophy
Sponsor: |
Isis Pharmaceuticals |
Enrolling: |
Male and Female Patients |
IRB Number: |
AAAO4103 |
U.S. Govt. ID: |
NCT02292537 |
Contact: |
Claudia Chiriboga: 212-305-8549 / cac3@columbia.edu |
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in limbs. Children will be treated with ISIS-396443 intrathecally for approximately 9 months followed by a 6-month post-treatment evaluation period.
This study is closed
Investigator
Claudia Chiriboga, MD, MPH
Has your child been diagnosed with spinal muscular atrophy (SMA)? |
Yes |
No |
Is your child able to sit independently, but does not have the ability to walk independently? |
Yes |
No |
Does you child have a history of bacterial meningitis? |
Yes |
No |