NCT06567769进行中（未招募）1 期
A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 9
- 试验地点
- 9
- 主要终点
- Incidences and characteristics of adverse events
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with documented MPS IIIA diagnosis
- •Participants aged ≥ 12 months and ≤ 18 years
排除标准
- •Participants with significant non-MPS IIIA related central nervous system impairment
- •Participants with previous complication from intraventricular drug administration
- •Participants with contraindications for MRI scans and for neurosurgery
- •Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study
- •Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
研究组 & 干预措施
Cohort 1
Experimental
干预措施: GC1130A (Drug)
Cohort 2
Experimental
干预措施: GC1130A (Drug)
Cohort 3
Experimental
干预措施: GC1130A (Drug)
结局指标
主要结局
Incidences and characteristics of adverse events
时间窗: up to 108 weeks
次要结局
- Maximum concentration in cerebrospinal fluid (CSF)(up to 104 weeks)
- Area under the concentration-time curve in CSF(up to 104 weeks)
- Maximum concentration in serum(up to 104 weeks)
- Area under the concentration-time curve in serum(up to 104 weeks)
- Change from baseline in CSF heparan sulfate concentration(up to 104 weeks)
- Change from baseline in serum heparan sulfate concentration(up to 104 weeks)
- Incidence of anti-drug and neutralizing antibodies of GC1130A in serum(up to 104 weeks)
- Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF(up to 104 weeks)
- Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)(up to 104 weeks)
研究者
研究点 (9)
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