[Translation] A double-blind, randomized, placebo-controlled trial evaluating the safety and efficacy of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) in the treatment of adults with spinocerebellar ataxia
主要目的:
确定 SLS-005 0.75 g/kg 治疗成人脊髓小脑性共济失调 3 型 (SCA3) 的疗效
次要目的:
确定 SLS-005 治疗成人脊髓小脑共济失调 3 型 (SCA3) 的安全性和耐受性
探索性目的:
评估 0.5 g/kg 的 SLS-005 治疗成人脊髓小脑性共济失调 3 型 (SCA3) 的疗效
确定 SLS-005 在 SCA 成人中的 PK
确定 SLS-005 治疗对与 SCA 成人神经轴突损伤相关的血液生物标志物的影响
[Translation] main purpose:
To determine the efficacy of SLS-005 0.75 g/kg in the treatment of adults with spinocerebellar ataxia type 3 (SCA3)
Secondary purpose:
To determine the safety and tolerability of SLS-005 in adults with spinocerebellar ataxia type 3 (SCA3)
Exploratory purpose:
To evaluate the efficacy of SLS-005 at 0.5 g/kg in the treatment of adults with spinocerebellar ataxia type 3 (SCA3)
Determining the PK of SLS-005 in adults with SCA
To determine the effect of SLS-005 treatment on blood biomarkers associated with axonal damage in adults with SCA