The STEER research study

Novartis believes all people with spinal muscular atrophy (SMA) – a rare, genetic, neuromuscular disease – should have access to gene therapy that addresses the root cause of their disease. 

This is one reason why we’re conducting the STEER clinical trial, a global research study of the intrathecal (IT) administration of OAV101, an investigational gene therapy. 

STEER is exploring the efficacy, safety, and tolerability of OAV101 IT in children and teens between 2 to <18 years of age who have SMA Type 2.¹

^{All compounds under investigation are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established for this new use. There is no guarantee that they will become commercially available for the use(s) under investigation.}

Introducing STEER

About the STEER study

STEER is a Novartis research study evaluating the clinical efficacy, safety and tolerability of OAV101 IT in patients with SMA Type 2 between 2 to <18 years of age who are able to sit but never walked, and have never received treatment for SMA. 

This research study is a randomized, double-blind, sham-controlled, global Phase 3 clinical trial.¹

In STEER, patients will be randomly assigned to one of two possible treatment groups, including:¹

• Treatment with OAV101 IT
• Sham procedure, a needle prick in the lower back without the administration of OAV101 IT

Eligible patients who have the sham procedure in the first period of the study will receive OAV101 IT in the second period of the study.

Spinal Muscular Atrophy

SMA: a rare genetic disease affecting around 1 in 10,000 births worldwide^4,5

• SMA is caused by the lack of a functional survival motor neuron 1 (SMN1) gene^6,7
• Untreated, SMA leads to irreversible loss of motor neurons, affecting breathing, swallowing, and basic movement in its most severe forms^6,7
• The severity of SMA varies across a spectrum of types (Type 1–4)⁸

The STEER research study focuses on Type 2.

SMA Type 2

• Accounts for approximately 30% of all SMA cases⁴
• Typically presents between 6 and 18 months of age⁴
• Patients learn to sit independently, but often lose this ability by their adolescent years⁹ 

Untreated, people with SMA Type 2 will never walk without support and will need a wheelchair or other mobility aids.¹⁰

How STEER works

Participating in the STEER study

Eligible patients who take part in the STEER study will receive:

• Investigational gene therapy OAV101 IT and a sham procedure
• Care and monitoring during the study
• Support with travel to and from the site where the study is conducted

Patients will be asked to stay in the STEER research study for about 15 months.  

The research study includes two treatment periods and two follow-up periods. If an eligible patient does not receive OAV101 IT in the first period, he or she will receive it in the second period. Similarly, eligible patients who receive OAV101 IT in the first treatment period, will receive sham procedure in the second period.¹

At the end of the research study, patients will be eligible to enroll in a long-term follow-up study to monitor long-term safety and efficacy.¹

Staying informed during the study

Caregivers and/or patients will be able to discuss any questions or concerns with the study doctors.

Learn more about the STEER research study.

Eligibility

Inclusion criteria: who can take part in the study?

To take part in the STEER research study, patients must:¹

• Be diagnosed with SMA Type 2
• Be between 2 to <18 years of age
• Have never received treatment with any SMN targeting therapies, such as risdiplam or nusinersen
• Be able to sit independently but have never walked independently

Additional inclusion and exclusion criteria apply. For more information about the STEER research study and key inclusion and exclusion criteria, visit the STEER research study page.