Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
Public ClinicalTrials.gov record NCT03306277. 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
Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
Study identification
- NCT ID
- NCT03306277
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 22 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Up to 180 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
- Oct 24, 2017 (Actual)
- Primary completion
- Nov 12, 2019 (Actual)
- Completion
- Nov 12, 2019 (Actual)
- Last update posted
- Jan 26, 2026
2017 – 2019
United States locations
- U.S. sites
- 16
- U.S. states
- 15
- U.S. cities
- 16
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| David Geffen School of Medicine at UCLA | Los Angeles | California | 90095 | — |
| Stanford University | Stanford | California | 94305 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Ann and Robert H Lurie Children's Hospital | Chicago | Illinois | 60611 | — |
| Johns Hopkins Pediatric Neurology | Baltimore | Maryland | 21287 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Washington Unviersity School of Medicine | St Louis | Missouri | 63110 | — |
| Columbia University | New York | New York | 10032 | — |
| Duke University | Durham | North Carolina | 27713 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Oregon Health and Science University | Portland | Oregon | 97239 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75235 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
| University of Wisconsin (Madison) | 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.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03306277, 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 26, 2026 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03306277 live on ClinicalTrials.gov.