A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies


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Trial ID: NCT02594124,25513,31500,26510


The primary objective is to evaluate the long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.

Official Title

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443

Stanford Investigator(s)

John W. Day, MD, PhD
John W. Day, MD, PhD

Professor of Neurology (Adult Neurology), of Pediatrics (Genetics) and, by courtesy, of Pathology


Key Inclusion Criteria:

   - Signed informed consent of parent or guardian and signed informed assent of
   participant, if indicated per participant's age and institutional guidelines.

   - Completion of the index study in accordance with the study protocol or as a result of
   Sponsor decision (e.g., early termination of the index study) within the preceding 16

Key Exclusion Criteria:

   - Have any condition or worsening condition which in the opinion of the Investigator
   would make the participant unsuitable for enrollment, or could interfere with the
   participant participating in or completing the study

   - Clinically significant abnormalities in hematology or clinical chemistry parameters or
   electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit
   that would render the participant unsuitable for participation in the study

   - Participant's parent or legal guardian is not willing or able to meet standard of care
   guidelines (including vaccinations and respiratory syncytial virus prophylaxis if
   available), nor provide nutritional and respiratory support throughout the study

   - Treatment with another investigational agent, biological agent, or device within one
   month of Screening, or 5 half-lives of study agent, whichever is longer

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.


drug: nusinersen


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Contact Information

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