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

Study ID
STU 012013-043

Cancer Related

Healthy Volunteers

Study Sites

  • Children's Medical Center-Dallas

Lauren Smith

Principal Investigator
Susan Iannaccone


This study will test the safety, tolerability, and pharmacokinetics of a single dose of iSiS 396443 (9 mg) administered as an intrathecal injection by lumbar puncture (LP).
after informed consent/assent is obtained, subjects will undergo a Screening evaluation no greater than 28 days prior to dose administration at which time their eligibility for the study will be examined. Subjects who meet the eligibility criteria will be admitted to the study center on Study Day 1, undergo pre-dose evaluations, and then receive an LP injection of study drug (9 mg iSiS 396443). The subject will remain at the study center for at least 6 hours post-injection for safety monitoring and laboratory specimen collection. Subjects will return to the study center on Study Day 8, 85, and 169 for follow-up evaluations. additionally, the study center will monitor the subject's condition through telephone contact on Study Day 2, 15, 29, 57, and 127.

Participant Eligibility

1. Signed informed consent of parent or guardian. Signed informed assent of subject, if indicated per patient[Single Quote]s age and institutional guidelines.
2. Satisfactory completion of dosing and all study visits in ISIS 396443-CS1 with an acceptable safety profile, per Investigator judgment
3. Able to complete all study procedures, measurements and visits and parent/patient has adequately supportive psychosocial circumstances, in the opinion of the Investigator
4. Estimated life expectancy >2 years from screening
5. Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedures (as assessed by the Site Investigator and either anesthesiologist or pulmonologist)
6. For subjects who, in the opinion of the Investigator, have reached reproductive maturity, satisfy one of the following:
Females: have a negative pregnancy test at Screening and must not be able to become pregnant (e.g., be abstinent or use adequate birth control methods as defined in Section 6.3) for the duration of the study
Males: be abstinent for the duration of the study