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NCT03381729 · ClinicalTrials.gov registry record · Phase 1
Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy
A Phase 1 study, sponsored by Novartis Gene Therapies.
- Terminated
- Registry status
- Phase 1
- Development phase
- 32
- Enrollment target
NCT03381729: Clinical Trial Phase 1 study, sponsored by Novartis Gene Therapies.
NCT03381729 is a Phase 1 study that was terminated before completion, run by Novartis Gene Therapies. The registered enrollment target is 32 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (47% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03381729, a Phase 1 study, was terminated before completion, sponsored by Novartis Gene Therapies.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 32 participants
- Enrollment target
Study Summary
The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
Primary Outcome
Defined by the Bayley Scales of Infant and Toddler Development (BSID) Gross Motor (GM) subtest performance criteria number 40, confirmed by video recording, as a participant who stands alone for at least 3 seconds unsupported.
Interventions
- BIOLOGICAL Onasemnogene Abeparvovec-xioi
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2017-12-14 |
| Est. Completion | 2021-11-18 |
| Phase | Phase 1 |
Why NCT03381729 stopped before completion
NCT03381729 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (47% lower).
The record links to 0 conditions, and to 1 intervention - of which Onasemnogene Abeparvovec-xioi is the first listed.
NCT03381729 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03381729 about?
NCT03381729 is a clinical study titled "Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy". The purpose of this trial is to evaluate the safety and tolerability of intrathecal administration of onasemnogene abeparvovec-xioi in infants and children with Spinal Muscular Atrophy with 3 copies of SMN2 and deletion of SMN1.
What is the current status of trial NCT03381729?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 32 participants. The study started on 2017-12-14. Estimated completion is 2021-11-18.
What interventions are being tested in trial NCT03381729?
The interventions under investigation include: Onasemnogene Abeparvovec-xioi (BIOLOGICAL).
Who is sponsoring clinical trial NCT03381729?
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 NCT03381729's enrollment target sits among peer trials
32 1296th of 2000 higher than 679 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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