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NCT03421977 · ClinicalTrials.gov registry record
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
A clinical trial of Spinal Muscular Atrophy 1, sponsored by Novartis Gene Therapies.
- Active
- Registry status
- 13
- Enrollment target
- 1
- Study location
NCT03421977: Active study of Spinal Muscular Atrophy 1, sponsored by Novartis Gene Therapies.
NCT03421977 is a study of Spinal Muscular Atrophy 1 that is active but no longer recruiting, run by Novartis Gene Therapies. The registered enrollment target is 13 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03421977, a study of Spinal Muscular Atrophy 1, is active but no longer recruiting, sponsored by Novartis Gene Therapies.
- ACTIVE NOT RECRUITING
- Registry status
- 13 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
Primary Outcome
The primary objective is to collect long term safety data of patients with SMA Type 1 who were treated with onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial by assessing incidence of SAEs and Adverse Events of Special Interest
Conditions Studied
Interventions
- BIOLOGICAL Onasemnogene Abeparvovec-xioi
Study Locations (1)
Ohio
- Nationwide Children's Hospital - Columbus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 13 participants |
| Start Date | 2017-09-21 |
| Est. Completion | 2030-12-02 |
What the registry record for NCT03421977 still lists
NCT03421977 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 13 participants, a relatively small participant target.
The record links to 1 condition, with Spinal Muscular Atrophy 1 appearing as the primary indexed condition, and to 1 intervention - of which Onasemnogene Abeparvovec-xioi is the first listed.
NCT03421977 reports a single indexed study location in Ohio.
Frequently Asked Questions
What is clinical trial NCT03421977 about?
NCT03421977 is a clinical study titled "Long-Term Follow-up Study for Patients From AVXS-101-CL-101". This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.
What is the current status of trial NCT03421977?
This trial is currently active not recruiting. The enrollment target is 13 participants. The study started on 2017-09-21. Estimated completion is 2030-12-02.
What conditions does trial NCT03421977 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy 1.
What interventions are being tested in trial NCT03421977?
The interventions under investigation include: Onasemnogene Abeparvovec-xioi (BIOLOGICAL).
Who is sponsoring clinical trial NCT03421977?
This trial is sponsored by Novartis Gene Therapies, which has 6 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03421977 being conducted?
This trial has 1 study location across Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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