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: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 24 Mar 2025

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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

21 Mar 2025 Planned End Date changed from 20 Feb 2025 to 29 Apr 2025.
19 Mar 2025 According to Novartis Media release, the data from this study presented during the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference held in Dallas, Texas, from March 16 to 19, 2025.
19 Mar 2025 Results published in the Novartis Media Release

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