Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial

Trial Name

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion.

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 six months of age with SMA Type 1.

What is involved?

48–72 hour stay in Nemours Children’s Hospital for the injection and 15 follow-up visits over 12 months. Procedures and tests to be done include a single injection by IV through arm or leg, ECG, echo, physical exam, blood and urine samples and various muscle tests.