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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

The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS1 (NCT02865109). The secondary objective was to examine the plasma pharmacokinetics of a single dose of ISIS 396443 administered intrathecally to participants with SMA 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 : nusinersen

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 (NCT01494701) with an acceptable safety profile, per Investigator judgement. - Able to complete all study procedures, measurements and visits and parent/participant 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 Key

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Contact information

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

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