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.

Investigator(s):

Intervention(s):

  • drug : ISIS-SMNRx

Phase: Phase 1

Eligibility

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

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Contact information

Primary Contact:

Shirley Paulose 650-724-3792

Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305

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