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NCT03306277 · ClinicalTrials.gov registry record · Phase 3
Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1
A Phase 3 study, sponsored by Novartis Gene Therapies.
- Completed
- Registry status
- Phase 3
- Development phase
- 22
- Enrollment target
NCT03306277: Completed Phase 3 study, sponsored by Novartis Gene Therapies.
NCT03306277 is a Phase 3 study that has completed, run by Novartis Gene Therapies. The registered enrollment target is 22 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03306277, a Phase 3 study, has completed, sponsored by Novartis Gene Therapies.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 22 participants
- Enrollment target
Study Summary
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
Primary Outcome
Independent sitting is defined as sitting up straight with head erect for at least 30 seconds. This endpoint is a co-primary endpoint. The two co-primary efficacy endpoints were assessed in sequence: The endpoint of functional independent sitting was assessed first and, only when this assessment met statistical significance, was the endpoint of event-free survival assessed.
Interventions
- BIOLOGICAL Onasemnogene Abeparvovec-xioi
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2017-10-24 |
| Est. Completion | 2019-11-12 |
| Phase | Phase 3 |
What the finished NCT03306277 record still lists
NCT03306277 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (97% lower).
The record links to 0 conditions, and to 1 intervention - of which Onasemnogene Abeparvovec-xioi is the first listed.
NCT03306277 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03306277 about?
NCT03306277 is a clinical study titled "Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1". Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.
What is the current status of trial NCT03306277?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 22 participants. The study started on 2017-10-24. Estimated completion is 2019-11-12.
What interventions are being tested in trial NCT03306277?
The interventions under investigation include: Onasemnogene Abeparvovec-xioi (BIOLOGICAL).
Who is sponsoring clinical trial NCT03306277?
This trial is sponsored by Novartis Gene Therapies, which has 6 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03306277's enrollment target sits among peer trials
22 1953rd of 2000 higher than 48 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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