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NCT03505099 · ClinicalTrials.gov registry record · Phase 3
Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2
A Phase 3 study, sponsored by Novartis Gene Therapies.
- Completed
- Registry status
- Phase 3
- Development phase
- 30
- Enrollment target
NCT03505099: Completed Phase 3 study, sponsored by Novartis Gene Therapies.
NCT03505099 is a Phase 3 study that has completed, run by Novartis Gene Therapies. The registered enrollment target is 30 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03505099, a Phase 3 study, has completed, sponsored by Novartis Gene Therapies.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 30 participants
- Enrollment target
Study Summary
To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
Primary Outcome
Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 26, confirmed by video recording, as a participant who sits for at least 30 seconds without assistance from another person or object. The participant was allowed to use their upper extremities.
Interventions
- BIOLOGICAL onasemnogene abeparvovec-xioi
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2018-04-02 |
| Est. Completion | 2021-06-15 |
| Phase | Phase 3 |
What the finished NCT03505099 record still lists
NCT03505099 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 30 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower).
The record links to 0 conditions, and to 1 intervention - of which onasemnogene abeparvovec-xioi is the first listed.
NCT03505099 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03505099 about?
NCT03505099 is a clinical study titled "Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2". To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2
What is the current status of trial NCT03505099?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 30 participants. The study started on 2018-04-02. Estimated completion is 2021-06-15.
What interventions are being tested in trial NCT03505099?
The interventions under investigation include: onasemnogene abeparvovec-xioi (BIOLOGICAL).
Who is sponsoring clinical trial NCT03505099?
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 NCT03505099's enrollment target sits among peer trials
30 1917th of 2000 higher than 68 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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