clinical trial · NCT04042025
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Spinal Muscular Atrophy Type ISpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type IIISMAOnasemnogene Abeparvovec-xioi
brief 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.
started
Feb 10, 2020
primary completion
Dec 31, 2035
completion
Dec 31, 2035
last updated
Apr 25, 2025
official title
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol