A Phase 3, Randomized, Single-blind, Parallel Group, Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Intracerebroventricular Tralesinidase Alfa Treatment Compared to Standard of Care in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 14
- 主要终点
- Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score
研究概览
简要总结
The primary objectives of this study are to evaluate the effects of Tralesinidase Alfa (TA) on cognition
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 1 Year 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity during screening.
- •Has nonattenuated severe MPS IIIB by the Genotype and Clinical Assessment Committee.
- •Is ≥1 and ≤5 years of age with BSID-III-C raw score of <
- •Is male or female as identified at birth.
排除标准
- •Genotyped for a known MPS IIIB variant associated with an attenuated phenotype or has a sibling with a known attenuated phenotype.
- •Has another neurological illness that may have caused cognitive decline (e.g., trauma, meningitis, or hemorrhage) before study entry.
- •Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.
研究组 & 干预措施
Tralesinidase alfa (TA)
干预措施: Tralesinidase alfa (TA) (Drug)
Control
结局指标
主要结局
Change from Baseline in Bayley Scales of Infant and Toddler Development, Third Edition, Cognition Domain (BSID-III-C) Raw Score
时间窗: Baseline to Week 260 (approximately 5 years)
The BSID-III-C is a standardized tool that evaluates cognitive development in young children, capturing skills such as processing speed, problem solving, and play. Raw scores are used to sensitively measure individual change over time, independent of age. Assessments are administered by a trained, blinded rater and reviewed by a central blinded rater. The endpoint is the difference in mean change from baseline in BSID-III-C raw score between the tralesinidase alfa and control arms at Week 260
次要结局
未报告次要终点