Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
Public ClinicalTrials.gov record NCT03505099. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
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
Study identification
- NCT ID
- NCT03505099
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Novartis Gene Therapies
- Industry
- Enrollment
- 30 participants
Conditions and interventions
Conditions
Interventions
- onasemnogene abeparvovec-xioi Biological
Biological
Eligibility (public fields only)
- Age range
- Up to 42 Days
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 1, 2018
- Primary completion
- Jun 14, 2021
- Completion
- Jun 14, 2021
- Last update posted
- Jan 25, 2026
2018 – 2021
United States locations
- U.S. sites
- 11
- U.S. states
- 11
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| David Geffen School of Medicine at UCLA | Los Angeles | California | 90095 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | — |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Clinic for Special Children | Strasburg | Pennsylvania | 17579 | — |
| Children's Medical Center Dallas | Dallas | Texas | 75235 | — |
| University Hospital and UW Health Clinics | Madison | Wisconsin | 53792 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03505099, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 25, 2026 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03505099 live on ClinicalTrials.gov.