A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT) - RESILIENT

Biohaven Pharmaceuticals, Inc0 个研究点目标入组 180 人开始时间: 2022年9月6日最近更新: 2024年7月15日

适应症

试验速览

暂无简介。

•Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number
•? Ambulant or non-ambulant
•? Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the trial, including the following:
•i. a stable regimen of nusinersen for 6 months
•prior to Screening; and/or
•ii. a stable regimen of risdiplam, for 6 months
•prior to Screening; and/or
•iii. a single dose of onasemnogene abeparvovec,
•received at least 2 years prior to Screening.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 153
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 27
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•? Receiving or have received previous administration of anti-myostatin
•? Weight <15 kg
•? Respiratory insufficiency, defined by the medical necessity for invasive or non-invasive ventilation for daytime treatment while awake (use overnight or during daytime naps is acceptable)
•? History of spinal fusion or major surgeries within 6 months prior to screening or planned during the study. Non-surgical adjustments are allowed during the study (such as MAGEC rods).
•? Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter

发起方