STEER Trial Results
STEER Trial Results
Page last updated: 6th January 2025
Novartis announces positive results of Phase III STEER Trial of OAV101 IT
This study evaluated the safety and efficacy of injecting the gene therapy onasemnogene abeparvovec (OAV101 IT) directly into the fluid bathing the brain and spinal cord (intrathecal delivery).
The study included treatment-naïve people between the ages of 2 and 17 with SMA Type 2, who could sit but had never walked independently.
More than 100 children were randomized to receive OAV101 by IT injection or to receive a sham procedure. This means one group receives a placebo .
At the end of the 52-week period, all eligible patients who received the sham procedure received OAV101 IT. All eligible patients who received OAV101 IT received the sham procedure.
A sham-controlled study would be unethical in countries where treatments are available. For this reason, this trial did not operate in the UK or mainland Europe. It was offered in countries where no treatments were available.
Key Findings:
OAV101 IT met its primary endpoint.
Compared to the sham-treated control group, participants treated with OAV101 IT showed a significant improvement in the Hammersmith Functional Motor Scale – Expanded (HFMSE). This is a test to measure muscle function and disease progression. This clinically meaningful improvement in motor function, builds on earlier findings from Phase I and II of the STRONG study.
A favourable safety profile was also demonstrated. Adverse events, including upper respiratory infections, fever and vomiting, were similar between the treatment and control groups. There were no major safety concerns.
Experts highlighted the potential of OAV101 IT to improve independence in daily activities, such as feeding and mobility, for older people living with SMA.
Next Steps:
Novartis plans to engage with regulatory agencies to make OAV101 IT available to people who live with SMA.
Further Information
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 medinfo.gtx@novartis.com
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.