2024-511852-42-00招募中3 期
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)
适应症
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 104
- 试验地点
- 21
- 主要终点
- Change from baseline in the MFM-32 at Week 48
研究概览
简要总结
To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable regimen of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec-xioi, compared to placebo, measured by change in the 32 item Motor Function Measure (MFM-32) total score between Baseline and Week 48.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •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 dose throughout the trial including nusinersen, risdiplam and/or history of onasemnogene abeparvovec
排除标准
- •Cannot have previously taken anti-myostatin therapies
- •Must weigh at least 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 within 6 months of Screening. MAGEC rod non-surgical adjustments are allowed during the study
- •Presence of an implanted shunt for the drainage of CSF or an implanted central nervous system (CNS) catheter
结局指标
主要结局
Change from baseline in the MFM-32 at Week 48
Change from baseline in the MFM-32 at Week 48
次要结局
- Change from baseline in the RULM at Week 48
- Change from baseline in the RHS at Week 48
- Safety and tolerability assessments including change in lean body mass and bone mineral density on DXA scan from baseline at Week 48 and Tanner staging for puberty monitoring, monitoring of injection acceptability assessments, and frequency of unique subjects with: new or worsening lab abnormalities, treatment related adverse events, serious adverse events, and adverse events leading to discontinuation.
- Trough plasma concentrations of taldefgrobep alfa and pharmacokinetic parameters estimated with population PK modeling
研究者
Lia Donahue
Scientific
Biohaven Pharmaceuticals Inc.
研究点 (21)
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