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Page last checked: 22nd November 2022
Page last updated: 22nd February 2022


This is a randomised, double-blind, sham-controlled study with a primary objective to evaluate the clinical efficacy, safety, and tolerability of a one-time intrathecal (IT) dose of OAV-101 in treatment naïve children and young people with Type 2 SMA who are between 2 and 18 years of age, able to sit, but have never walked.

More than 100 children and young people will be randomised to receive OAV-101 by IT injection or to receive a sham procedure. At the end of the 52-week period, all eligible participants who received the sham procedure will receive OAV-101 IT and all eligible patients who received OAV-101 IT will receive the sham procedure.

The therapeutic effect of OAV-101 IT will be evaluated using a test to measure motor function, the Hammersmith Functional Motor Scale-Expanded (HFMSE).

Secondary objectives include evaluation of safety and the efficacy of one-time IT administration of OAV-101 using a test to measure upper limb function, the Revised Upper Limb Module (RULM) scale.

A sham-controlled study – which means one groups receives a placebo – would be unethical in countries where treatments are available. For this reason, this trial won’t operate in the UK or mainland Europe. It will be offered in countries where no treatments are available.

Questions & Answers from the Novartis Gene Therapies Team

STEER will build upon promising data from the Phase 1/2 STRONG study, which to date has shown that treatment with OAV-101 IT led to significant increases in HFMSE scores and a clinically meaningful response. We believe that investigational OAV-101 IT is a viable potential treatment path for older patients with SMA and for whom a single-dose, treatment option could be especially compelling.

Study plans for STEER are still being finalised. For more information about the study, contact the Novartis Gene Therapies Medical Information team at

It is important to clarify that enrolment is not anticipated to start until later this year.

The use of a sham control offers an optimal chance of obtaining clear data that would allow for approval to treat more segments of the community.

STEER will build upon the Phase 1/2 STRONG study which showed that treatment with OAV-101 IT led to significant increases in HFMSE scores and a clinically meaningful response in older patients >2 years and <5 years old with SMA Type 2.