Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
Public ClinicalTrials.gov record NCT05089656. 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 Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory
Study identification
- NCT ID
- NCT05089656
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Enrollment
- 126 participants
Conditions and interventions
Conditions
Interventions
- OAV101 Genetic
- Sham control Procedure
Genetic · Procedure
Eligibility (public fields only)
- Age range
- 2 Years to 17 Years
- 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
- Jan 31, 2022
- Primary completion
- Nov 11, 2024
- Completion
- Apr 28, 2025
- Last update posted
- Jan 12, 2026
2022 – 2025
United States locations
- U.S. sites
- 6
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Connecticut Children's Medical Center | Farmington | Connecticut | 06032 | — |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | — |
| Clinic for Special Children | Strasburg | Pennsylvania | 17579 | — |
| St Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
| Child Hosp Of The Kings Daughters | Norfolk | Virginia | 23507 | — |
| Children's Specialty Group/CHKD | Norfolk | Virginia | 23507 | — |
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 36 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05089656, 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 12, 2026 · Synced May 16, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05089656 live on ClinicalTrials.gov.