Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Phase IIIb open-label single-arm IT OAV101 in pediatric SMA previously treated with nusinersen or risdiplam (Japan)
jRCT2073220094Phase 3Completed
- Sponsor
- Novartis
- Start
- Jan 2023
- Modality
- Gene Therapies
01
Overview
Phase 3, open-label, single-arm trial of onasemnogene abeparvovec in spinal muscular atrophy (SMA), sponsored by Novartis. Primary endpoint: Safety: adverse events. Started Jan 2023; status: completed.
02
Drugs
onasemnogene abeparvovec
03
Conditions
spinal muscular atrophy (SMA)
Beyond the preview
Go deeper on jRCT2073220094.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on jRCT2073220094.