Clinical Trials Directory

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 to see if other locations are recruiting.



  • drug : ISIS-SMNRx

Phase: Phase 1


Ages Eligible For Study:

2 Years - 15 Years

Inclusion Criteria

- 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

External Links

Explore related trials

Contact information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Shirley Paulose
Not Recruiting

Footer Links: