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
A study for patients with spinal muscular atrophy using study drug, ISIS 396443 to test safety and effectiveness
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
Additional Study Information: 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
Do You Qualify?
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
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Place Holder




For more information, please contact:
Claudia Chiriboga
cac3@columbia.edu
212-305-8549