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DOI: 10.1055/s-0039-1698171
Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe
Publication History
Publication Date:
11 September 2019 (online)
Research Question: SMA1, a rapidly progressing disease, typically results in death/need for permanent ventilation by 2 years of age. Survival motor neuron 1 gene (SMN1) biallelic deletion/mutation is the genetic root cause of SMA and leads to irreversible motor neuron loss. Onasemnogene abeparvovec (AVXS-101) is an investigational, one-time GRT based on a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human SMN gene. AVXS-101 is designed for immediate and sustained SMN protein expression, allowing rapid onset and durable effect in neurons. In a phase 1/2a study (CT.gov: NCT02122952), SMA1 patients treated with AVXS-101 demonstrated exceptional improvements in survival, motor function, bulbar function, and milestone achievement. STR1VE-EU is a phase 3, multicenter, open-label, single-arm, one-time-dose study (EudraCT: 2017-000266-29; CT.gov: NCT03461289) investigating efficacy and safety of AVXS-101 in up to 30 SMA1 patients
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No conflict of interest has been declared by the author(s).