[Translation] A Phase II, multicenter, randomized, double-blind, placebo-controlled study (followed by an open-label extension phase) to evaluate the efficacy and safety of SAR443820 in adult subjects with amyotrophic lateral sclerosis
主要目的:
A 部分
评估SAR443820与安慰剂相比在减缓ALS进展方面的作用(通过修订版肌萎缩侧索硬化功能评分量表[ALSFRS-R]测定)。
B部分
评估SAR443820对功能和生存期的长期影响。
次要目的:
A部分
评估SAR443820与安慰剂相比对功能和生存期的综合评估、呼吸功能、肌肉力量和生活质量(QoL)的影响;评估SAR443820与安慰剂相比对关键疾病生物标志物的药效学(PD)效应;评估SAR443820与安慰剂相比的安全性和耐受性;评估SAR443820的药代动力学(PK)。
B部分
评估SAR443820对疾病进展、生存期、呼吸功能和生活质量(QoL)的长期影响;评估SAR443820对关键疾病生物标志物的长期影响;评估SAR443820的长期安全性和耐受性;评估SAR443820的药代动力学(PK)。
[Translation] Primary objectives:
Part A
To evaluate the effect of SAR443820 compared with placebo in slowing ALS progression (measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised [ALSFRS-R]).
Part B
To evaluate the long-term effects of SAR443820 on function and survival.
Secondary objectives:
Part A
To evaluate the effects of SAR443820 compared with placebo on a composite assessment of function and survival, respiratory function, muscle strength, and quality of life (QoL); to evaluate the pharmacodynamic (PD) effects of SAR443820 compared with placebo on key disease biomarkers; to evaluate the safety and tolerability of SAR443820 compared with placebo; and to evaluate the pharmacokinetics (PK) of SAR443820.
Part B
Evaluate the long-term effects of SAR443820 on disease progression, survival, respiratory function, and quality of life (QoL); evaluate the long-term effects of SAR443820 on key disease biomarkers; evaluate the long-term safety and tolerability of SAR443820; and evaluate the pharmacokinetics (PK) of SAR443820.