A Phase lllb, open-label, single-arm, single-dose, multicenter study to evaluate the safety, tolerability and efficacy of gene replacement therapy with intravenous OAV101 (AVXS-101) in pediatric patients with spinal muscular atrophy (SMA) - SMART

ovartis Pharma AG0 个研究点目标入组 24 人开始时间: 2021年6月28日最近更新: 2022年5月30日

适应症

试验速览

暂无简介。

•Symptomatic SMA diagnosis based on gene mutation analysis with bi-allelic survival motor neuron 1 (SMN1) mutations (deletion or point mutations) and any copy of the survival motor neuron 2 (SMN2) gene.
•Weight = 8.5 kg and = 21 kg at the time of Screening Visit 2
•Naive to treatment or have discontinued an approved drug/therapy
•other protocol-defined inclusion criteria may apply
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 24
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Previous OAV101 use or previous use of any AAV9 gene therapy
•Body Mass Index (BMI) < 3rd percentile based on World Health Organization (WHO) Child Growth Standard
•Participant with history of aspiration pneumonia or signs of aspiration (eg, coughing or sputtering of food) within 4 weeks prior to screening
•Anti-Adeno-associated virus serotype 9 (AAV9) antibody titer > 1:50 as determined by ligand binding immunoassay at the time of screening
•History of gene therapy, hematopoietic transplantation, or solid organ transplantation
•other protocol-defined exclusion criteria may apply

发起方