Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Nonambulatory Patients

Trial Name

Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting but Non-ambulatory Patients with Spinal Muscular Atrophy.

What is the trial about?

The purpose of this study is to find out if this one-time therapy can replace the SMN protein with one that is fully functional.

Who can participate?

Children under five years of age with SMA Type 1.

What is involved?

48–72 hour stay in Nemours Children’s Hospital for the spinal injection and 15 follow-up visits over 12 months. Procedures and tests to be done include a single injection by lumbar puncture, EKG, physical exam, blood and urine samples and various muscle tests.