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NCT05089656 · ClinicalTrials.gov registry record · Phase 3
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
A Phase 3 study, sponsored by Novartis Pharmaceuticals.
- Completed
- Registry status
- Phase 3
- Development phase
- 126
- Enrollment target
NCT05089656: Completed Phase 3 study, sponsored by Novartis Pharmaceuticals.
NCT05089656 is a Phase 3 study that has completed, run by Novartis Pharmaceuticals. The registered enrollment target is 126 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (84% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05089656, a Phase 3 study, has completed, sponsored by Novartis Pharmaceuticals.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 126 participants
- Enrollment target
Study Summary
This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.
Primary Outcome
The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
Interventions
- GENETIC OAV101
- PROCEDURE Sham control
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 126 participants |
| Start Date | 2022-02-01 |
| Est. Completion | 2025-04-29 |
| Phase | Phase 3 |
What the finished NCT05089656 record still lists
NCT05089656 is an interventional study that assigns participants to a tested intervention. The registered 126 participants enrollment target is mid-sized for trials with a published cap, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (84% lower).
The record links to 0 conditions, and to 2 interventions - of which OAV101 is the first listed.
NCT05089656 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT05089656 about?
NCT05089656 is a clinical study titled "Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)". This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.
What is the current status of trial NCT05089656?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 126 participants. The study started on 2022-02-01. Estimated completion is 2025-04-29.
What interventions are being tested in trial NCT05089656?
The interventions under investigation include: OAV101 (GENETIC), Sham control (PROCEDURE).
Who is sponsoring clinical trial NCT05089656?
This trial is sponsored by Novartis Pharmaceuticals, which has 1,375 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT05089656's enrollment target sits among peer trials
126 1603rd of 2000 higher than 396 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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