SMN2遺伝子を1つまたは2つ有する脊髄性筋萎縮症1型患者に対する単回投与遺伝子置換療法

基本情報

NCT ID

NCT03837184

ステータス

完了

試験のフェーズ

第3相

試験タイプ

介入

目標被験者数

2

治験依頼者名

Novartis

概要

This is a Phase 3, open-label, single-arm, single-dose, trial of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 and who are genetically defined by a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). The primary objective of the study is to evaluate the efficacy of onasemnogene abeparvovec-xioi by assessing the proportion of symptomatic SMA Type 1 participants who achieve the ability to sit unaided for at least 10 seconds up to and including the 18 months of age trial visit. At least 6 participants aged \< 6 months (\< 180 days) at the time of gene replacement therapy (Day 1) will be enrolled.

対象疾患

介入

依頼者（Sponsor）

実施施設 (1)