Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
Public ClinicalTrials.gov record NCT03381729. 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 I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
Study identification
- NCT ID
- NCT03381729
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 1
- Enrollment
- 32 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Months to 60 Months
- 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
- Dec 14, 2017 (Actual)
- Primary completion
- Nov 18, 2021 (Actual)
- Completion
- Nov 18, 2021 (Actual)
- Last update posted
- Jan 26, 2026
2017 – 2021
United States locations
- U.S. sites
- 11
- U.S. states
- 10
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCLA | Los Angeles | California | 90095 | — |
| Stanford University | Stanford | California | 94305 | — |
| Nemours Children's Hospital | Orlando | Florida | 32827 | — |
| Ann & Robert H. Lurie Children's Hospital | Chicago | Illinois | 60611 | — |
| Johns Hopkins | Baltimore | Maryland | 21287 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Washington University | St Louis | Missouri | 63130 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| UT Southwestern | Dallas | Texas | 75390 | — |
| University of Utah | Salt Lake City | Utah | 84112 | — |
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 NCT03381729, 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 22, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03381729 live on ClinicalTrials.gov.