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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 is a Phase 1 study that was terminated before completion, run by Novartis Gene Therapies. The registered enrollment target is 32 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03381729
The ClinicalTrials.gov registry entry for NCT03381729 describes a study currently listed as terminated, categorized as Phase 1. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Novartis Gene Therapies, which has 6 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Onasemnogene Abeparvovec-xioi is the first listed.
NCT03381729 reports 0 study locations.
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.
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