A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA
Latest Information Update: 06 Jun 2024
At a glance
- Drugs Onasemnogene-abeparvovec (Primary)
- Indications Spinal muscular atrophy
- Focus Adverse reactions
- Acronyms OFELIA
- Sponsors Novartis Pharmaceuticals
- 30 Aug 2023 Status changed from active, no longer recruiting to completed.
- 18 Jul 2023 Planned End Date changed from 30 Jun 2023 to 31 Jul 2023.
- 18 Jul 2023 Planned primary completion date changed from 30 Jun 2023 to 31 Jul 2023.