Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

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

Next Previous
Trial Profile

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

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 12 Dec 2025

Price :
$35 *
Note:
  • Adis is an information provider.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our trial profile at the time of purchase.
  • A link to download a PDF version of the trial profile will be included in your email receipt.

At a glance

  • Drugs Onasemnogene abeparvovec (Primary) ; Prednisolone sodium phosphate
  • Indications Spinal muscular atrophy
  • Focus Registrational; Therapeutic Use
  • Acronyms STEER
  • Sponsors Novartis Gene Therapies; Novartis Pharma A.G.; Novartis Pharmaceuticals

Most Recent Events

  • 24 Nov 2025 According to Novartis Media release, based on data from the registrational Phase lll STEER study and supported by the open-label Phase lllb STRENGTH study, US Food and Drug Administration (FDA) has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA).
  • 04 Jul 2025 Status changed from active, no longer recruiting to completed.
  • 21 Mar 2025 Planned End Date changed from 20 Feb 2025 to 29 Apr 2025.

You need to be a logged in or subscribed to view this content

Request demo (opens in a new window)
If your organization or you do not have a subscription, try one of the following: If your organization has a subscription, there are several access options, even while working remotely:
  • Working within your organization’s network
  • with username/password or try to via your institution
  • Persisted access using your organization’s identifier stored in your user browser for 90 days
Back to top