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STEER: Clinical Trial on Use of Intrathecal Zolgensma (OAV101) to Treat Spinal Muscular Atrophy in Children and Teens

About this study

The STEER study aims to find out if an injection of the gene therapy OAV101 (Zolgensma®) into the spinal cord is safe and benefits children with SMA. Doctors will use a thin needle to put OAV101 into the lower back. The drug is injected into the fluid-filled space below the end of the spinal cord.

Children in the study will be admitted to the hospital. They will get either OAV101 or a placebo. Then they will be followed for 1 year. At the end of the year, they will enter the hospital again. Then they will get either the OAV101 or the placebo, whichever they did not get during the first admission.

Purpose of this clinical trial

The purpose of the STEER study is to find out if an injection of the OAV101 into the spinal fluid is safe and benefits children with SMA who are between ages 2 and 18.

Eligibility overview

  • Ages 2 years to 17 years
  • Patients with spinal muscular atrophy (SMA)
  • Never treated for SMA with medications such as Spinraza or Evrysdi, or previously treated with Zolgensma
  • Able to sit, but never able to walk

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.


Full title:

A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

Study goal:

To investigate the safety, tolerability, and efficacy of the gene therapy OAV101 (Zolgensma®) delivered intrathecally in treatment-naïve SMA Type 2 patients between ages 2 and 18 with 2-4 copies of SMN2.


2 years to 17 years

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.


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