An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1
This study will test the safety and tolerability of ISIS-SMNRx administered into the spinal fluid as a single injection in patients with Spinal Muscular Atrophy, who previously participated in ISIS 396443-CS1
Stanford is not currently accepting new patients for this trial. You may want to check clinicaltrials.gov to see if other locations are recruiting.
- drug : ISIS-SMNRx
Phase: Phase 1
Ages Eligible For Study:
- Clinical signs attributable to Spinal Muscular Atrophy - Satisfactory completion of dosing and all study visits in ISIS 396443-CS1 with an acceptable safety profile, per Investigator judgement. - Able to complete all study procedures, measurements and visits and parent/patient has adequately supportive psychosocial circumstances, in the opinion of the investigator - Estimated life expectancy > 2 years from Screening - Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedure