Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
OFELIA
2023-000864-67Phase 4
- Sponsor
- Novartis
- Start
- Feb 2024
- Modality
- Gene Therapies, Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoints: Treatment-emergent adverse events and serious adverse events over 18 months, Important identified and potential risks and 1 more.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
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