Trials / Active Not Recruiting
Active Not RecruitingNCT04042025
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 85 (actual)
- Sponsor
- Novartis Gene Therapies · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
Summary
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Onasemnogene Abeparvovec-xioi | Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight. |
Timeline
- Start date
- 2020-02-10
- Primary completion
- 2035-12-31
- Completion
- 2035-12-31
- First posted
- 2019-08-01
- Last updated
- 2025-04-25
Locations
31 sites across 9 countries: United States, Australia, Belgium, Canada, France, Italy, Japan, Taiwan, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04042025. Inclusion in this directory is not an endorsement.