オナセムノゲンアベパルボベック-xioi投与患者の長期追跡調査

基本情報

NCT ID

NCT04042025

ステータス

実施中（募集終了）

試験のフェーズ

第3相

試験タイプ

介入

目標被験者数

85

治験依頼者名

Novartis

概要

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.

対象疾患

介入

依頼者（Sponsor）

実施施設 (1)