Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Public ClinicalTrials.gov record NCT04042025. 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 Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
Study identification
- NCT ID
- NCT04042025
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 85 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- 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
- Feb 10, 2020 (Actual)
- Primary completion
- Dec 31, 2035 (Estimated)
- Completion
- Dec 31, 2035 (Estimated)
- Last update posted
- Sep 24, 2026
2020 – 2035
United States locations
- U.S. sites
- 18
- U.S. states
- 15
- U.S. cities
- 17
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford University Medical Center | Palo Alto | California | 94304 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | — |
| Ann Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| John Hopkins Hospital - David M. Rubenstein Child Health Building | Baltimore | Maryland | 21287 | — |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | — |
| Spectrum Health Hospitals Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | — |
| Washington Unviersity School of Medicine in Saint Louis | St Louis | Missouri | 63110 | — |
| Columbia University Medical Center | New York | New York | 10032 | — |
| Duke University | Durham | North Carolina | 27713 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Clinic for Special Children | Strasburg | Pennsylvania | 17579 | — |
| Children's Health Specialty Center Dallas Campus | Dallas | Texas | 75235 | — |
| University of Utah Health | Salt Lake City | Utah | 84112 | — |
| Children's Hospital of The King's Daughters | Norfolk | Virginia | 23507 | — |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | — |
| 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.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 13 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04042025, 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 Sep 24, 2026 · Synced Oct 20, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04042025 live on ClinicalTrials.gov.