Clinical trial
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal…
NCT03505099 · Novartis Gene Therapies, Industry · Completed
Key facts
| NCT number | NCT03505099 |
|---|---|
| Title | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 |
| Official title | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2 |
| Acronym | SPR1NT |
| Lead sponsor | Novartis Gene Therapies, Industry |
| Collaborators | PRA Health Sciences Industry |
| Status | Completed |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Spinal Muscular Atrophy |
| Intervention type | Biological |
| Interventions | onasemnogene abeparvovec-xioi |
| Enrollment | 30 |
| Start date | Apr 2, 2018 |
| Primary completion | Jun 15, 2021 |
| Completion | Jun 15, 2021 |
| First posted | Apr 23, 2018 |
| Last update posted | Jan 26, 2026 |
| Countries | Australia, Belgium, Canada, Japan, United Kingdom, United States |
At a glance
- 10–99 Study sites
- Yes Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: