Long-Term Follow-up Study for Patients From AVXS-101-CL-101
Public ClinicalTrials.gov record NCT03421977. 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 Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101
Study identification
- NCT ID
- NCT03421977
- Recruitment status
- Active, not recruiting
- Study type
- Observational
- Phase
- Not listed
- Lead sponsor
- Novartis Gene Therapies
- Industry
- Enrollment
- 13 participants
Conditions and interventions
Conditions
Interventions
- Onasemnogene Abeparvovec-xioi Biological
Biological
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
- Sep 20, 2017
- Primary completion
- Dec 1, 2030
- Completion
- Dec 1, 2030
- Last update posted
- Apr 24, 2025
2017 – 2030
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
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 NCT03421977, 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 Apr 24, 2025 · Synced May 16, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03421977 live on ClinicalTrials.gov.